CAMBRIDGE, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, announced today that it will highlight its ADAR (adenosine deaminases acting on RNA)-mediated RNA editing technology (ADAR editing) and its neurology programs in Huntingtons disease (HD), amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) at upcoming conferences, including the TIDES: Oligonucleotide and Peptide Therapeutics 2020 meeting and the 16th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), taking place on September 15-18, 2020 and September 27-30, 2020, respectively.

At TIDES and OTS, Wave will share how it is developing ADAR editing as a potentially best-in-class RNA editing approach with applicability across multiple genetic diseases. Notably, one of Waves presentations at TIDES will include the companys first ADAR editing data in ex vivo retina samples from non-human primates (NHPs). These data add to the growing body of in vitro and in vivo data the company has generated, which include editing in the liver of NHPs and the central nervous system in a humanized mouse model, in support of ADAR editing as an emerging platform capability at Wave.

Another presentation at TIDES will highlight Waves neurology pipeline, including its C9orf72 variant-selective silencing program for ALS and FTD, as well as its SNP3 program for HD, both of which are expected to initiate clinical development with the submission of clinical trial applications in the fourth quarter of 2020.

Details of Data Presentations

TIDES: Oligonucleotide and Peptide Therapeutics 2020 meeting

16th Annual Meeting of the Oligonucleotide Therapeutics Society

About PRISMPRISM is Wave Life Sciences proprietary discovery and drug development platform that enables genetically defined diseases to be targeted with stereopure oligonucleotides across multiple therapeutic modalities, including silencing, splicing and editing. PRISM combines the companys unique ability to construct stereopure oligonucleotides with a deep understanding of how the interplay among oligonucleotide sequence, chemistry and backbone stereochemistry impacts key pharmacological properties. By exploring these interactions through iterative analysis ofin vitroandin vivooutcomes and machine learning-driven predictive modeling, the company continues to define design principles that are deployed across programs to rapidly develop and manufacture clinical candidates that meet pre-defined product profiles.

About Wave Life Sciences Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the companys proprietary discovery and drug development platform that enables the precise design, optimization and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit http://www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the development of ADAR editing as a potentially best-in-class RNA editing approach and emerging platform capability at Wave; the potential for Waves preclinical data, including data from and its neurology programs in Huntingtons disease (HD), amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD); and the expected timing for the initiation of clinical development for Waves C9orf72 variant-selective silencing program for ALS and FTD, as well as its SNP3 program for HD. The words may, will, could, would, should, expect, plan, anticipate, intend, believe, estimate, predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, the risks and uncertainties described in the section entitled Risk Factors in Waves most recent Annual Report on Form 10-K filed with theSecurities and Exchange Commission(SEC), as amended, and in other filings Wave makes with theSECfrom time to time. Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances

Investor Contacts:Kate Rausch617-949-4827krausch@wavelifesci.com

Graham Morrell781-686-9600gmorrell@wavelifesci.com

Media Contact:Alicia Suter617-949-4817asuter@wavelifesci.com

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Wave Life Sciences to Highlight Preclinical ADAR Editing Data and Neurology Programs at TIDES and OTS Annual Meetings - GlobeNewswire

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--ESCAPE Bio, a clinical stage company developing novel, precisely targeted therapeutics for genetic neurodegenerative diseases, today announced the closing of a $73M financing led by Wellington Management Company LLP. Additional new investors include Avidity Partners, CAM Capital, New Leaf Ventures, Rock Springs Capital, Surveyor Capital (a Citadel company) and Sphera Funds Management, who join existing investors OrbiMed, Novo Holdings, Johnson & Johnson Innovation, Novartis Venture Fund, Osage University Partners and Sutter Hill Ventures.

We are delighted to partner with these new outstanding investors as we advance our pipeline of precision neurology medicines, commented Julie Anne Smith, President and Chief Executive Officer of ESCAPE. The proceeds allow us to accelerate two programs into patients who lack disease modifying treatments.

About ESCAPE Bio

ESCAPE Bio is a clinical stage, privately held biopharmaceutical company developing novel, precisely targeted therapeutics for genetically defined neurodegenerative diseases. ESB1609 is in a Phase 1 multiple ascending dose study in healthy volunteers. ESCAPE has advanced a mutant-selective LRRK2 G2019S kinase inhibitor for Parkinsons Disease (PD) patients to IND-enabling studies. A pharmacologic structure corrector of Alzheimer's patients carrying the ApoE4 risk allele is in Discovery. For additional information, please visit http://www.escapebio.com.

About ESB1609

ESB1609 is a novel, orally administered, brain-penetrant and selective sphingosine-1-phosphate 5 (S1P5) receptor agonist. S1P5 receptors are predominantly expressed in the central nervous system (CNS) and natural killer (NK) cells as one of five receptors within the G-protein-coupled S1P receptor family (S1P1 S1P5). Activation of S1P5 plays a significant counteractive role in many aspects of neurodegenerative disease relevant biology inclusive of upregulating several CNS lipid transporters. S1P5 agonism has normalized brain ceramide and sphingosine phosphate levels and promoted clearance of aggregation-prone proteins across multiple pre-clinical models of neurodegeneration. ESB1609 is currently in a Phase 1 randomized, double-blind, placebo-controlled, safety, tolerability, pharmacokinetic and biomarker study of escalating multiple doses in healthy volunteers.

About ESB5070

ESB5070 is a novel, orally-administered, brain-penetrant and selective Leucine-Rich Repeat Kinase 2 (LRRK2) G2019S kinase inhibitor that spares wild type LRRK2 functionality. G2019S is the most common pathogenic mutation linked to PD occurring in 13% of PD patients. G2019S drives elevated kinase activity that perturbs the delicate orchestration of downstream signaling required for the healthy functioning of multiple cellular processes. ESB5070 is being developed specifically for the treatment of subjects carrying the LRRK2 G2019S variant and is currently in IND-enabling toxicology studies.

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ESCAPE Bio Closes $73 Million in Crossover Financing to Advance Clinical Development of Precision Neurology Medicines for Genetic Neurodegeneration -...

ASPEN It isnt noticeable at first. It starts with changes for which the brain can compensate, meaning no real impact on day-to-day functions or cognition.

But as time passes, the brain can no longer compensate for the damage its experiencing. Subtle problems with memory and thinking begin to pop up. Subtle turns to noticeable. Noticeable turns to difficulty carrying out everyday activities. Eventually, around-the-clock care is required.

This is the broad view progression from preclinical to severe Alzheimers disease, a degenerative brain disease that becomes worse with time and age, and is the most common cause of dementia, according to the Alzheimers Association.

In Colorado, an estimated 76,000 people are living with Alzheimers dementia, and that number is expected to increase 21% to 92,000 by 2025, a 2020 Alzheimers Association report states. As of July 2019, 14.6% of Coloradans, or about 840,000 people, were 65 or older, U.S. Census Bureau data shows.

While its been shown that living in higher elevation communities can lead to a more active, healthier lifestyle and even prolonged life, its less clear how living at high elevation correlates with degenerative brain diseases.

In short, the answer is complicated and not well researched.

As far as I know, there isnt a lot of evidence one way or another about high altitudes versus low altitudes for Alzheimers disease risk, said Dr. Huntington Potter, director of the Alzheimers and Cognition Center at the University of Colorado Anschutz Medical Campus. We cant say one way or another whether high altitude is a risk factor for Alzheimers.

At the Alzheimers and Cognition Center, which is part of the CU Anschutz Medical Campus and School of Medicine, clinicians and researchers are dedicated to discovering effective early diagnostics, preventions, treatments and ultimately cures for Alzheimers disease and related neurodegenerative disorders, according to its website.

For Potter, that means looking at biomarkers or diagnostic proteins in the blood that can help clinicians predict the disease earlier, conducting projects that look at the lifespan of people with Alzheimers disease, and other research that can quickly be translated to better care, treatment and hopefully a cure.

Right now, the center is studying a drug called Leukine, which preliminary data shows might improve Alzheimers disease in the short term, Potter explained. The center also is studying other drugs that attack the disease.

Leukine may be the first one we found that looks promising, but we have several coming up that look promising, as well, Potter said.

When it comes to looking at the potential correlation between living at high elevation and the risk for dementia-inducing diseases, Potter and Dr. Peter Pressman of the Alzheimers and Cognition Center said it would take great effort, time and funding to research.

Assistant professor for the Department of Neurology, Dr. Peter S. Pressman works in his office at the University of Colorados Anschutz Medical Campus in Aurora on Thursday, Sept. 10. Photo by Liz Copan / Studio Copan

Assistant professor for the Department of Neurology, Dr. Peter S. Pressman is pictured outside his office building at the University of Colorados Anschutz Medical Campus in Aurora on Thursday, Sept. 10.Photo by Liz Copan / Studio Copan

Assistant professor for the Department of Neurology, Dr. Peter S. Pressman works in his office at the University of Colorados Anschutz Medical Campus in Aurora on Thursday, Sept. 10. Photo by Liz Copan / Studio Copan

Assistant professor for the Department of Neurology, Dr. Peter S. Pressman works in his office at the University of Colorados Anschutz Medical Campus in Aurora on Thursday, Sept. 10. Photo by Liz Copan / Studio Copan

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Pressman, who is a behavioral neurologist and researcher with the center, said on top of securing and carefully selecting a large group of people living at elevation to participate in a study, researchers also would have to follow that group for about a decade to get meaningful results.

Its easy to fund a study for a few years, Pressman said. People give you money to do something for two to three years, but two to three years is not enough time for a process as slow as Alzheimers and dementia to really even pick up. Im not saying thats not possible. Its doable, but it would take some effort.

Dr. Brooke Allen, neurologist, founder of Roaring Fork Neurology in Basalt and medical director at Renew Roaring Fork, an assisted living and memory care center in Glenwood Springs, expressed similar thoughts.

As a part of any mild cognitive impairment or dementia evaluation, Allen said her team checks the oxygen level a patient has and considers the elevation at which that person spends most of their time.

Lower oxygen levels can contribute to people experiencing confusion, dizziness and mild short-term memory issues. But outside of looking at oxygen levels and how they could be contributing to symptoms, Allen said she doesnt consider elevation a higher risk situation.

Allen said she feels High Country residents 65 and older tend to be much younger than their age in terms of their lifestyle, which is a positive in terms of dementia prevention.

About four years ago, Allen and her team conducted a long-term preclinical Alzheimers trial as part of the Alzheimers Prevention Initiatives Generation Program, a study that looked at the effectiveness of preventative treatments for individuals between 65 and 75 who had no symptoms of dementia.

Allen said more than 250 people came in to participate in the study and all generally led healthy, active lifestyles.

I think in our valley, Ive experienced meeting those kinds of people and not thinking of altitude as a risk factor but as a lifestyle opportunity in a rural area like ours, Allen said about the people who participated in the study.

Looking at the potential correlation between living at high elevation and risk of degenerative brain diseases is not just a difficult feat for Colorado researchers. Little research with concrete findings exists nationally or globally.

One study published in 2015 by Dr. Stephen Thielke in JAMA Psychiatry looked at deaths attributed to Alzheimers dementia reported in 58 counties in California to try to determine whether rates of dementia were associated with average elevation of residence. The study found that the counties at higher elevation generally had lower rates of dementia mortality.

Additional work is needed to determine whether this relationship holds in other populations, the study notes.

But beyond this study, there isnt much conclusive evidence for or against a correlation, as emphasized by Dr. Brent Kious, a psychiatrist, assistant professor and researcher with University of Utah Health and the schools Department of Psychiatry.

Kious has studied the link between living at high elevations and major depressive disorder, anxiety and suicide, and he said he and his research team have been interested in the impact of elevation on the incidence and median age of onset of Parkinsons disease.

However, Kious said decrements in cognitive performance due to chronic exposure to moderately high elevation might not necessarily translate into an increased risk of dementia.

It is not clear whether altitude would affect those neurodegenerative processes or not, though there is some reason to think that they involve oxidative damage so relative hypoxia might slow them, Kious wrote in an email. He went on to note that relative and prolonged hypoxia, or a lack of oxygen, has been associated with dementia risk. In any case, a good epidemiological study of the association between altitude and dementia should control for things that might be associated with both.

Karen Eck, 60, of Silverthorne, picks tomatoes at the Timberline Adult Day Program garden in Frisco on Thursday, Sept. 10. Eck, who was diagnosed with early onset Alzheimers, attends programs three to four times a week at the center.Photo by Jason Connolly / Jason Connolly Photography

Karen Eck, 60, of Silverthorne, waters tomatoes at the Timberline Adult Day Program garden in Frisco on Thursday, Sept. 10. Eck, who was diagnosed with early onset Alzheimers, attends programs three to four times a week at the center.Photo by Jason Connolly / Jason Connolly Photography

Karen Eck, 60, of Silverthorne, poses for a portrait at the Timberline Adult Day Program garden in Frisco on Thursday, Sept. 10. Eck, who was diagnosed with early onset Alzheimers, attends programs three to four times a week at the center.Photo by Jason Connolly / Jason Connolly Photography

Karen Eck, 60, of Silverthorne, enters the Timberline Adult Day Program in Frisco on Thursday, Sept. 10. Eck has early onset Alzheimers.Photo by Jason Connolly / Jason Connolly Photography

Karen Eck, 60, of Silverthorne, leaves the Timberline Adult Day Program garden in Frisco after watering tomatoes on Thursday, Sept. 10. Jason ConnollyPhoto by Jason Connolly / Jason Connolly Photography

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While theres not good data for or against high elevation as a risk factor for degenerative brain diseases, there is evidence that people living in more rural communities do not have the same access to dementia care and treatment as those living in urban areas.

According to the 2020 Snapshot of Rural Health in Colorado, produced by the Colorado Rural Health Center, 721,500 people are living in rural Colorado and 19% of the rural population is age 65 or older. Rural is defined as a nonmetropolitan county with no cities over 50,000 residents.

Chad Federwitz a gerontologist, or specialist in the study of aging, and manager of Pitkin County senior services said he hasnt seen any correlation between living at high elevation and dementia risk. Anecdotally, he does know that people move to Grand Junction or the Front Range if they have dementia because of a lack of care resources in the High Country.

Given the nature of our rural-ish community, we dont have the same resources, Federwitz said, referring to things like long-term assisted living and memory care options. You can go to Grand Junction or the Front Range and have pages and pages of resources as opposed to here.

While there are some dementia care resources in more rural Colorado communities, the Alzheimers and Cognition Center is working to do more to develop meaningful relationships with health care providers and dementia patients in the states mountain communities as part of its mission.

According to Pressman, who is heading this charge on behalf of the CU center, a lot of projects are in the planning stages but include virtually educating medical providers, nurse practitioners and primary care doctors on Alzheimers and general healthy brain aging as well as mutual, participatory research with rural Colorado communities and communities of color.

Pressman explained that a lot of research related to Alzheimers overwhelmingly is based on middle-class, well-educated, white participants. And so while researchers think they know a lot about the disease in general, they really only know about the disease related to this demographic group.

Through the centers outreach and efforts to better connect with underrepresented communities, Pressman hopes to conduct better science and better serve the larger Colorado community.

What motivates me is trying to do good work, trying to do good science and to make sure our results actually represent real life, Pressman said. We want to make sure were helping everybody, not just a niche group, and that our services are available equitably to as many people as possible.

Editors note: This is Part 2 of a four-part series on longevity in the High Country. The series is being produced in partnership with The Aspen Times, Glenwood Springs Post Independent, Steamboat Pilot & Today and Vail Daily. Read more at SummitDaily.com/longevity.

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The aging brain: Exploring the connection between neurology and elevation - Summit Daily News

Single-center prospective study evaluated feasibility of bedside MR neuroimaging in the intensive care settings for 50 critically-ill patients with COVID-19, stroke, hemorrhage, traumatic brain injury and tumors.

A recent study published in the Journal of the American Medical Association Neurology demonstrates for the first time the feasibility of Hyperfines Swoop Portable MRI System to obtain bedside neuroimaging for critically-ill patients in the intensive care unit settings. Researchers tested the feasibility and demonstrated the clinical utility of the Swoop system in 50 patients, including COVID-19 patients, to assess brain injury and detect abnormalities. These patients were critically ill and presented challenges for transport to conventional MRI suites in the Radiology Department.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200909005243/en/

Hyperfines Swoop Portable MRI System successfully detects abnormalities at bedside of critically-ill patients in intensive care unit settings. (Photo: Business Wire)

Results from the prospective, observational single-center study, Assessment of Brain Injury Using Portable, Low Field Magnetic Resonance Imaging at the Bedside of Critically Ill Patients, were published on September 8, 2020.

Investigators used the Swoop system from October 2019 through May 2020 and successfully detected abnormal neuroimaging findings at the bedside of patients presenting with ischemic stroke, hemorrhagic stroke, subarachnoid hemorrhage, traumatic brain injury, brain tumor and COVID-19 patients with altered mental status.

According to the studys senior author, Kevin N. Sheth, MD (Yale University School of Medicine, Department of Neurology), the use of traditional MRI units presents accessibility and transportation challenges in the care of critically-ill patients. Furthermore, the highly contagious nature of COVID-19 patients makes bedside MR imaging compelling for infection control protocols.

This study the first of its kind demonstrates that deployment of portable MR imaging to patients' bedsides could fill an important gap for time-sensitive neuroimaging, a cornerstone of triage and treatment pathways.

The study authors conclude, "This experience demonstrates that low-field, portable MRI can be deployed successfully into intensive care settings. This approach may hold promise for portable assessment of neurological injury in other scenarios, including the emergency department, mobile stroke units, and resource-limited environments."

Magnetic Resonance Imaging uses a magnetic field, radio waves and a computer to produce detailed pictures of the body's internal structures that are clearer, more detailed and more likely in some instances to identify and accurately characterize disease than other imaging methods. However, fixed MRI systems can be inconvenient and inaccessible for providers and patients, particularly when time is critical. Transport to the MR suite demands complicated scheduling coordination, moving patients, and, often, 4 to 6 hour patient backlogs all which compromise the utility of MRI as a diagnostic tool in time-sensitive settings such as intensive care units and emergency rooms. Furthermore, high capital investments, electrical power needs and significant maintenance requirements present a barrier to adoption across all populations, acutely so for developing countries and rural geographies.

Story continues

About the Swoop Portable MRI System

Hyperfines Swoop system was designed to address the limitations of current imaging technologies and make MRI accessible anytime, anywhere, to any patient. Swoop wheels directly to the patients bedside, plugs into a standard electrical wall outlet, and is controlled by a wireless tablet such as Apple iPad. Images are captured at the patients bedside, with results in minutes, enabling critical decision-making capabilities across a variety of clinical settings including neuro intensive care units, emergency departments, pediatrics, ambulatory outpatient surgery centers and more. The complete Hyperfine system costs less than the annual service contract alone for most current MRI systems, and it consumes 35 times less power than those same systems. Designed as a complementary system to traditional MRIs, new users can be trained on system operation, device navigation and device safety in about 30 minutes, helping clinicians to streamline workflow.

About Hyperfine Research

Hyperfine lives to make MR imaging available to everyone. The Swoop Portable MR Imaging System is the result of a total rethink of MRs potential in the healthcare landscape. What if MR imaging didnt require a dedicated suite, extensive training or expensive upkeep? Lets use the wonders of high-field MRI for the appropriate patients, and deploy Swoop system at the point-of-care for everyone else. Hyperfine received market-ready FDA clearance for its portable MR imaging for the brain and head of patients of all ages in August 2020. Hyperfine is part of 4Catalyzer, a health technology incubator with facilities in CT, NY, CA and Taiwan. http://www.hyperfine.io.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200909005243/en/

Contacts

Chris Ward, cward@hyperfine.io, (203) 905-0412

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Study Published in JAMA Neurology Confirms Low-Field MR Imaging Using Hyperfines Swoop Portable MRI Successfully Detects Abnormalities at Bedside of...

The following article is part of conference coverage from the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event. Neurology Advisors staff will be reporting breaking news associated with research conducted by leading experts in neurology. .

While both recognize the importance of a telemedicine approach, patients with multiple sclerosis (MS) appear more prepared and ready to use telemedicine than their physicians during the coronavirus disease 2019 (COVID-19) pandemic, according to study results presented at the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event, held September 11-13, 2020.

During the COVID-19 era, patients and clinicians alike increased their use of telemedicine platforms, particularly for routine evaluations that do not require in-person office visits. Study researchers sought to understand the acceptability and satisfaction of telemedicine among patients with MS and the neurologists who care for them.

This study consisted of a brief survey which was administered to a group of patients with multiple sclerosis and their neurologists at an MS center of a hospital in Milan, Italy. 151 patients completed the survey. Approximately 75% of the patient cohort consisted of women. The mean age of participants was 42.2 years, and the median Expanded Disability Status Scale was 1.5. Treatments included interferon (10%), glatiramer acetate (10%), teriflunomide (14%), dimethylfumarate (22%), fingolimod (23%), cladribine (3%), alemtuzumab (8%), and ocrelizumab (10%).

87% of patients said that they appreciated telemedicine during the COVID-19 pandemic. Contrastingly, responses from 82% supported traditional in-office evaluations and their importance in clinical care. Less than half (44%) of participants said that they would alternate in-person and remote telemedicine visits, whereas 38% strongly preferred traditional evaluations. About 10% held a positive opinion of telemedicine but required traditional evaluations. Only 3% of respondents were not satisfied with telemedicine.

The primary reasons given for the strong preference toward in-person evaluations included the need for human empathy with the neurologist, as well as the belief that in-person examinations would lead to better clinical outcomes. Only 18% of respondents said that they would always use telemedicine except if an acute event occurred.

None of the 18 neurologists and residents surveyed said that they would use telemedicine as their only tool for evaluating patients. One-third (33%) of clinicians said that they would alternate telemedicine visits with traditional evaluations, whereas 67% said they would use remote visits only in special situations.

Visit Neurology Advisors conference section for continuous coverage from the ACTRIMS/ECTRIMS MSVirtual2020 Forum.

Reference

Moiola L, Cristinzi MD, Guerrieri S, et al. Telemedicine in/outside the pandemic: a survey about satisfaction of this tool in a cohort of multiple sclerosis patients and their neurologists. Presented at: 8th Joint American Committee for Treatment and Research in Multiple Sclerosis and European Committee for Treatment and Research in Multiple Sclerosis MSVirtual2020 event; September 11-13, 20120. Abstract P0666.

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Patients With MS More Ready to Use Telemedicine Than Their Neurologists - Neurology Advisor

The following article is part of conference coverage from the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event. Neurology Advisors staff will be reporting breaking news associated with research conducted by leading experts in neurology. .

Among patients with multiple sclerosis (MS), a history of migraine may not be associated with worse disability or neurological function, according to study results presented at the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event, held September 11-13, 2020.

While previous studies reported that migraine is common in patients with MS, limited data exist on the effects of migraine on disability or neurological function in this population. The objective of the current study was to investigate the association between history of migraine, disability and neurological function in patients with MS. In addition, study researchers explored the association between migraine and frequency of MS relapses, and the co-occurrence of migraine with other comorbidities in patients with MS.

This observational study included patients with MS and a documented history of migraine, who completed a neurological function assessment using the MS Performance Test. Study researchers also collected data on other comorbidities, including diabetes, hypertension, dyslipidemia, history of myocardial infarction, sleep apnea, depression, and anxiety.

Disability was determined according to the Patient Determined Disease Steps tool. Additional outcomes included annualized relapse rate, rate of new brain lesions on MRI and objective neurological outcomes (walking speed, manual dexterity, and processing speed).

Of the 2017 patients with MS who completed the MS Performance Test, 336 had 1 documented diagnosis of migraine in their electronic medical record. Patients with MS and migraine were younger than those without migraine (mean age, 42.6 years vs 46.6 years, respectively; P <.001) and had higher rates of depression (46.52 vs. 48.16, P <.001), anxiety (50.29 vs 52.81; P <.01), and obstructive sleep apnea (109 vs 53, P <.001).

Severe disability was less frequent in patients with migraine compared to those without (5.4% vs 12%, respectively; P <.003), and there was no difference in objective neurological outcomes, including walking speed, manual dexterity or processing speed. Furthermore, there was no significant difference in annualized relapse rate or rate of new brain lesions in patients with MS with and without migraine.

Researchers concluded that a history of migraine was not associated with greater disability in patients with MS, but added that the evidence to date on this topic is conflicting and warrants future longitudinal studies..

Visit Neurology Advisors conference section for continuous coverage from the ACTRIMS/ECTRIMS MSVirtual2020 Forum.

Reference

Damian A, Hu C, Fitzgerald K, Mowry E. A history of migraine headache may not be associated with worse disability or worse neurological function. Presented at: 8th Joint American Committee for Treatment and Research in Multiple Sclerosis and European Committee for Treatment and Research in Multiple Sclerosis MSVirtual2020 event; September 11-13, 2020. Abstract P0423.

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Effects of Migraine on Disability and Neurological Function in MS - Neurology Advisor

The following article is part of conference coverage from the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event. Neurology Advisors staff will be reporting breaking news associated with research conducted by leading experts in neurology. .

For youths with pediatric multiple sclerosis (MS), activities and participation are more important life domains than disease-related impairments. As a result,

a health-related quality of life (HRQoL) measure should include variables of MS-related impairments that require targeted therapies, as well as the activities and roles important to these patients, according to study results presented at the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event, held September 11-13, 2020.

While generic HRQoL measures are used to evaluate the impact of pediatric MS, these measures do not always provide insight into domains of health that are relevant to children and parents alike. The goals of this study were to identify domains of life significant to youths with pediatric MS and to contribute to building a better condition-specific measure for this population.

To achieve these, study researchers used an online survey based on the Patient Generated Index (PGI), an approach which relies on open-ended responses to assess HRQoL. They then mapped text threads formed by the PGI onto the International Classification of Functioning, Disability and Health (ICF), and the Comprehensive ICF Core Set for MS.

A total of 19 participants completed the survey, 10 of whom were youths with MS between the ages of 14 and 22 years. This group had a median onset age of 13.5 years. More than 80% of the areas in the survey nominated by youths with MS were associated with activities and participation, while just 20% of these areas related to impairments. 62% of the areas nominated by parents, however, related to impairments. Taken together, these findings suggest that a HRQoL measure should include variables of MS-related impairments that require targeted therapies as well as activities and roles important to youths.

The researchers noted that the new assessment approach has a disability component which addresses such impairments related to MS, and is completed with the PGI system. In this component, the youth or parent chooses a total of 5 disability areas impacted by MS, rates its severity, and prioritizes each area for improvement. Additionally, the assessment has a youth-completed quality of life component that asks respondents about areas going well.

Study researchers concluded that the use of this new measurement approach could prove useful in overcoming challenges of measurement development for children with rare diseases like pediatric MS.

Visit Neurology Advisors conference section for continuous coverage from the ACTRIMS/ECTRIMS MSVirtual2020 Forum.

Reference

Ow N, Karp A, Ogeil J, Dilenge M, Sbire G, Mayo N. Pediatric MS: different disease course, different impact, different measurement approach needed. Presented at: 8th Joint American Committee for Treatment and Research in Multiple Sclerosis and European Committee for Treatment and Research in Multiple Sclerosis MSVirtual2020 event; September 11-13, 2020. Abstract P0130.

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Improvements Are Needed to Measure Impact of Pediatric MS - Neurology Advisor

FROM THE ACADEMY OF CONSULTATION-LIAISON PSYCHIATRY

CASE REPORT

Ms Zorn was a 45-year-old woman with no previous psychiatric history who presented with a 2-month period of feeling like I am losing myself and seeing visually distorted faces. Faces of people she was looking at would appear to be hideously elongated, with the facial components distorted, as if it was melting. Cars would also appear to be more round than usual.

She was admitted to an inpatient psychiatry unit for unspecified psychosis, but after 2 days she was transferred to a general medical unit for additional medical evaluation. She was started on 25-mg quetiapine bid, which was titrated to 200 mg bid. Lumbar puncture was completed to rule out central nervous system infection or autoimmune encephalopathy, with results pending at the time of transfer to an academic medical center for higher level of multispecialty care.

On interview, she said that she felt like she was losing herself and reported visual distortions of faces melting. Ms Zorn was able to recognize faces and colors. She reported photophobia. She had headaches, which were relieved with ketorolac. Her husband reported that she had new onset of episodes of shaking her arms in a circular motion. She would also walk around the room in circular patterns while saying to herself that she was lost.

Ms Zorn had decreased appetite, weight loss, and decreased sleep. She denied depressed mood, decreased energy, mania, trauma, or previous problems with memory. She denied auditory hallucinations and suicidal/homicidal ideation. She denied a family history of suicide and psychiatric illness. Developmental history was unremarkable. She had no history of substance abuse.

On mental status examination, she was awake, alert, oriented to person, time, place, and situation. She continued to experience visual distortions of faces melting. Ms Zorns thought process was linear and coherent. Affect was mildly anxiously perplexed, non-labile, and non-tearful. Speech was normal. Insight and judgment were marginal. Her Montreal Cognitive Assessment (MoCA) score was 10/30. Her decisional capacity was impaired, and the screening neurological examination was non-focal.

Due to the atypical presentation of psychotic disorder, the full workup included a neurology consultation; brain MRI and lumbar puncture results were normal, except for autoimmune encephalitis results that were pending. Serum HIV, syphilis, vitamin B12, lead, rapid plasma regain (RPR), anti-nuclear antibody ammonia, and thyroid stimulating hormone (TSH) levels were normal; EEG was unremarkable. Pelvic ultrasound and CT were ordered to rule out ovarian teratoma. Quetiapine regimen was changed to 100 mg in the morning and 300 mg in the evening.

Ms Zorn was seen in follow-up on hospital day 2. Neurology had started her on 1-g methylprednisolone daily for a 5-day course for presumed autoimmune encephalitis. She was placed on a continuous EEG. On exam, she reported feeling better compared with the previous day. She no longer reported any distortions of faces or objects in the room, although she did report some blue dots in her field of view. She had 3 hours of sleep the previous night. Her husband noted that the she had decreased episodes of her aimless pattern of waving her arms in a circular motion. Her MoCA score was 14/30. Due to the significant improvement in her hallucinations following the first dose of methylprednisolone, the scheduled quetiapine was held.

When seen on hospital day 3, Ms Zorn said that she could fall asleep, but had trouble staying asleep. She had an episode where she got up and walked around the room purposelessly. Her MoCA score was 13/30 and decisional capacity was impaired. Quetiapine 50 mg post merdien (PM) was started to address poor sleep. On hospital day 4, she reported that her visual phenomena had returned, with her seeing pixels (flashing punctate lights) and faces melting. She had had difficulty sleeping. MoCA was 12/30 with continued limited insight, judgment, and decisional capacity. Quetiapine was increased to 100 mg pm and mirtazapine 7.5 mg pm was started to improve sleep.

On hospital day 5, her husband reported that she had had continued visual phenomena of pixels and faces melting. Ms Zorn was only able to sleep for 1.5 hours after receiving quetiapine and mirtazapine. On exam, she reported the visual hallucinations and had a MoCA score of 12/30. Continuous EEG was reported as normal and abdomen CT was negative for ovarian teratoma. To address continued psychosis and poor sleep, quetiapine was increased to 100 mg am and 300 mg pm. Mirtazapine was increased to 15 mg pm.

Ms Zorn was seen again on hospital day 6. She reported that she had been able to sleep for 8 hours. She denied any visual melting of faces but stated that the world looked pixelated at times. MoCA score was 14/30. Subsequently, when seen on hospital day 8, she stated that she was still seeing pixels in the periphery of her visual field. MoCA score improved to 17/30. Quetiapine was consolidated to 400 mg pm.

She was next seen on hospital day 10. The cerebrospinal (CSF) encephalopathy panel came back positive for anti-NMDA receptor antibody R1, confirming a diagnosis of anti-NMDA (N-methyl-D-aspartate)-receptor encephalopathy (ANMDARE). Neurology started plasmapheresis for 7 treatments, every other day. She had no hallucinations. She reported that she was getting deep sleep. MoCA score was 16/30. A quetiapine 50 mg am dose was added.

Plasmapheresis started on hospital day 11. The patient denied any visual distortions. She said that she felt significantly better than the previous week; MoCA score was 16/30. When next seen on hospital day 15, Ms Zorn reported sleeping from 11:00 pm to 5:30 am. She denied visual distortions. MoCA score had improved to 21/30; however, decisional capacity continued to be impaired. On hospital day 18, she was doing well. She had declined her scheduled quetiapine the previous day to give it a try. She no longer saw distorted faces. She had no gait disturbance. MoCA score improved further to 25/30. Given her improved mental status, the psychiatry team decided that she had recovered her decision-making capacity. Mirtaza-pine was decreased to 7.5 mg pm and quetiapine held.

Ms Zorn was seen for the last time on day 22 of hospital admission. After her final plasmapheresis treatment, she continued to be free of psychotic symptoms. She stated that she was doing better than last week, and that she was 80% back to normal. She was sleeping well and was increasingly physically active during the day. She was excited to return home; MoCA score was 22/30. Decisional capacity continued to be intact. Mirtazapine 7.5 mg bedtime was continued.

As she was medically stable for discharge, she was referred for psychiatric follow-up near her home. She was advised that any future recurrence of psychosis should first be addressed as if it was a recurrence of delirium and/or ANMDARE.

Discussion

ANMDARE is a relatively recently described illness that may present with psychiatric symptoms, neurologic symptoms, or both (either simultaneously or sequentially).1-5 While the exact mechanism remains obscure, antibodies to the NMDA receptor (on CSF and/or serum assay) is confirmatory.2 Management includes empiric treatment of manifest symptoms and immunomodulation systemic therapies.4,5

ANMDARE is important to the consultation-liaison psychiatrist for a number of reasons. First, its presentation lies in the neuropsychiatric borderland involving collaboration between psychiatry and neurology (as in other neuropsychiatric illnesses like Parkinson disease, Huntington disease, and multiple sclerosis). Second is its importance in the often hard-to-define syndrome of atypical psychotic disorder. In comparison to the classic presentation of schizophrenia, ANMDARE features a later age of onset, female predominance, visual hallucinations, relative lack of cognitive disorganization, and a lack of an extended prodromal period.

This case illustrates the atypical presentation of psychotic illness rather well. Ms Zorn was in her mid-40s, of high academic and professional achievement, adaptive social function; without a clear prodrome, she relatively promptly developed a psychotic picture with striking and disturbing visual hallucinatory phenomena. Clinical suspicion for ANMDARE was confirmed with reference laboratory (Mayo Clinic) results of + R1 antibody to NMDA receptor on CSF analysis. This was accomplished in the context of a thorough laboratory evaluation for other esoteric causes of atypical psychotic illness.

Empiric treatment of the psychiatric symptoms with mirtazapine and quetiapine, concurrent with 2 rounds of immunomodulation therapy, led to amelioration of symptoms and near complete cognitive recovery in a period of 2.5 weeks. Her cognitive status based on objective measure with the Montreal Cognitive Assessment (MoCA) was initially in the moderate to severely impaired range.6 With comprehensive treatment, Ms Zorns cognitive status improved to a level of only mild impairment. Correspondingly, her decisional capacity improved from significantly impaired to largely intact over the same period.

Psychiatrists assessing and treating patients with atypical presentation of psychotic illness should actively consider a diagnosis of ANMDARE as explanatory.3,4 In female patients with suspicion of ANMDARE, pelvic ultrasonography and/or CT scan should be ordered to rule out commonly co-occurring ovarian teratoma.1 Patients with ANMDARE who are treated with high-dose intravenous corticosteroids should be monitored (and treated for) any corticosteroid-associated psychiatric adverse effects, which can overlap with the atypical psychotic symptoms at illness onset. Empiric treatment with psychopharmacology for psychotic and/or depressive symptoms should be pursued, although such medications may not be necessary indefinitely once definitive immunomodulation therapy is completed.4

ANMDARE should be on the differential for atypical psychosis, with an expectation of a thorough search for laboratory and systemic clinical (especially neurologic) findings, empiric treatment of psychiatric symptoms, and reassessment of the patient as immunomodulation therapy is completed. Prospective study of ANMDARE illness cohorts are needed to quantify recurrence risk, need for ongoing intervention, and ultimate prognosis.

Consultation-liaison psychiatrists working in academic medical centers may be in a position to assist multispecialty teams in the identification, diagnosis, management, and ongoing follow-up of these patients. As with any illness that affects cognitive function, attention to and serial assessment of decisional capacity is a critical part of C-L psychiatry care, especially as pertains to patient consent for major immunomodulation therapies.

Dr Bourgeois is chair, Department of Psychiatry, Baylor Scott & White Health, Central Texas Division, and clinical professor for medical education, Texas A&M University Health Science Center, Temple, TX. Mr Li is a medical student, Texas A&M University Health Science Center, Temple, TX. The authors report no conflicts of interest concerning the subject matter of this article.

References

1. Tuzun E, Zhou L, Baehring JM, et al. Evidence for antibody-mediated pathogenesis in anti-NMDAR encephalitis associated with ovarian teratoma. Acta Neuropathol. 2009;118:737-743.

2. Gresa-Arribas N, Titulaer MJ, Torrents A, et al. Antibody titres at diagnosis and during follow-up of anti-NMDA receptor encephalitis: a retrospective study. Lancet Neurol. 2014;13:167-177.

3. Kayser MS, Titulaer MJ, Gresa-Arribas N, Dalmau J. Frequency and characteristics of isolated psychiatric episodes in anti-N-methyl-d-aspartate receptor encephalitis. JAMA Neurol. 2013;70:1133-1139.

4. Warren N, OGorman C, McKeon G, et al. Psychiatric management of anti-NMDAR encephalitis: a cohort analysis. Psychol Med. November 19, 2019; Epub ahead of print.

5. Titulaer MJ, McCracken L, Gabilondo I, et al. Treatment and prognostic factors for long-term outcome in patients with anti-NMDA receptor encephalitis: an observational cohort study. Lancet Neurol. 2013;12:157-165.

6. Nasreddine Z. Montreal Cognitive Assessment (MoCA) Administration and Scoring Instructions. Montreal Cognitive Assessment. Accessed July 9, 2020. http://www.mocatest.org/wp-content/uploads/2015/tests-instructions/MoCA-Instructions-English_2010.pdf .

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Anti-NMDA Receptor Encephalitis With Visual Hallucinations and Cognitive Impairment - Psychiatric Times

The following article is part of conference coverage from the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event. Neurology Advisors staff will be reporting breaking news associated with research conducted by leading experts in neurology. .

Disease modifying therapies (DMTs) show the greatest effectiveness among those with multiple sclerosis (MS) who have shorter MS duration, lower scores on the Expanded Disability Status Scale (EDSS), lower rate of relapse, and the relapsing MS phenotype, according to research presented at the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event, held September 11-13, 2020.

This retrospective study included 26,329 individuals with relapsing or progressive MS selected from the international MSBase registry. Inclusion criteria consisted of being followed for 1 year, with 3 visits, and 1 visit per year. Study researchers compared the hazard ratios (HR) of EDSS improvement and 6-month confirmed worsening, as well as relapse rates between treated and untreated periods. Marginal structural models were used to do so and were continuously readjusted to take into account participant sex, age, date, pregnancy, previous relapse history, time from first symptom, MRI activity, and disability.

Among 23,687 individuals with relapsing MS, those treated with DMTs were 20% more likely to experience disability improvement (HR 1.20; 95% CI, 1.0-1.5), 47% less likely to experience worsening disability (HR 0.53; 95% CI, 0.39-0.71), and had a 51% decrease in relapses (HR 0.49; 95% CI, 0.43-0.55). However, longer MS duration and higher previous rate of relapse reduced the effect of DMTs on relapses and EDSS worsening; the effect of DMTs on these factors was stronger among those in lower EDSS categories.

The use of DMTs was associated with greater EDSS improvement among those without new MRI lesions (HR 1.51; 95% CI, 1.00-2.28) than those with MRI activity (HR 1.04; 95% CI, 0.88-1.24). Among the 26,329 individuals with either relapsing or progressive MS, those with relapsing MS treated with DMTs showed reductions in both EDSS worsening (HR 0.75; 95% CI, 0.65-0.86) and relapses (HR 0.58; 95% CI, 0.54-0.62). However, these benefits were not observed in those with progressive MS.

The study researchers concluded, DMTs are associated with reduction in relapse frequency, progression of disability, and increased chance of recovery from disability. however, subgroups with shorter MS duration, lower EDSS, lower relapse rate and relapsing MS phenotype appear more likely to experience benefits from DMTs.

Visit Neurology Advisors conference section for continuous coverage from the ACTRIMS/ECTRIMS MSVirtual2020 Forum.

Diouf I, Malpas C, Horakova D, et al. Variability of the response to immunotherapy among sub-groups of patients with multiple sclerosis. Presented at: 8th Joint American Committee for Treatment and Research in Multiple Sclerosis and European Committee for Treatment and Research in Multiple Sclerosis MSVirtual2020 event; September 11-13, 2020. Abstract P0018.

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DMTs Most Effective Among Certain Subgroups of Individuals With MS - Neurology Advisor

An exclusive Interventional Neurology Market research report has been fabricated through the in depth analysis of the market dynamics across five regions including North America, Europe, South America, Asia-Pacific, Middle East and Africa.

The segmentation of the market by components, end users, and region was done based on the thorough market analysis and validation through extensive primary inputs from industry experts (key opinion leaders of companies, and stakeholders) and secondary research (global/regional associations, trade journals, technical white papers, companys website, annual report SEC filing, and paid databases). Further, the market has been estimated by utilizing various research methodologies and internal statistical model.

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The research dives deep into the global share, size, and trends, as well as growth rate of the Interventional Neurology Market to project its progress during the forecast period, i.e., 20202027. Most importantly, the report further identifies the past, present, and future trends that are expected to influence the development rate of the Interventional Neurology Market. The research segments the market on the basis of product type, application, and region.

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Interventional Neurology Market Estimated to Expand at a Robust CAGR by 2027 - The Market Correspondent

Featured Image: (L-R) Barbara Johnson, Elizabeth Matthews and Mikisha Johnson. When Elizabeth Matthews (center, pictured with daughter Barbara Johnson, left, and Mikisha Johnson, right) had a stroke, doctors discovered she also had COVID-19. From strokes to mental health, the Emory Brain Health Center is leading research on the neurological effects of the pandemic.

By Emory University

Mikisha Johnson hung up the phone. On the call, her 83-year-old grandmother, Elizabeth Matthews, had struggled to string words together and sounded disoriented.

Grandmama doesnt sound right, Mikisha told her mother, Barbara.

When Barbara Johnson arrived the following morning at her parents home in the southwest Atlanta neighborhood of Collier Heights, her mother didntlookright either.

I said Mama, youre slurring your words, your mouth is twisted and your hand is trembling, Barbara Johnson said. You are going to the hospital.

Elizabeth Matthews already had a pretty good idea what was wrong, but she didnt want to worry her daughter.

I said to my husband, I believe I had a stroke, she recalled.

She was right. But that wasnt all. At that visit in early August, doctors at Emory St. Josephs Hospital told Matthews shed also tested positive for COVID-19.

I got real emotional when they told me that, Matthews said. I was thinking, Am I going to die?

Since the COVID-19 outbreak began late last year, it has largely been understood as an assault on the respiratory system. Telltale symptoms are often a fever, hacking cough and difficulty breathing; patients in the worst shape end up on respirators.

What is still less understood, but just as alarming, is the damage the virus may be doing to the brain, from strokes like Matthews to reports of headaches, seizures and confusion. And that doesnt even take into account the staggering toll of the pandemic on our mental health.

Today, more than 300 studies from around the world have looked at links between neurological problems and COVID-19. More are underway.

We are now recognizing COVID-19 disease actually has a significant neurological implication or neurologic effect, said Byron Milton III, MD, a physiatrist, or physical medicine and rehabilitation doctor, at Emory University Hospital who has helped COVID patients cope with dementia-like symptoms and other neurological problems.

(L-R) Barbara Johnson, Elizabeth Matthews and Mikisha Johnson. Elizabeth Matthews (center, with daughter Barbara Johnson, left, and granddaughter Mikisha Johnson, right) battled back from stroke and COVID-19. Shes looking forward to a new grandchild, due in late September.

Even as they care for patients, researchers and health care providers at the Emory Brain Health Center are among those leading the way toward understanding the short- and long-term neurological implications of the pandemic on the brain and the mind.

Those efforts are featured in Season 2 of the Your Fantastic Mind television series from Georgia Public Broadcasting and Emory University, which debuted Sept. 9 and continues through Oct. 14.

One of the things that really sets Emory apart is the multi-disciplinary way the Brain Health Center works, said Jonathan Lewin, MD, Emorys executive vice president for health affairs and executive director of the Woodruff Health Sciences Center.

Emory combines neurology, psychiatry and behavioral sciences, neurosurgery, rehabilitation medicine and sleep medicine. Thats proving to have a real benefit during this pandemic, where were learning so much about the virus every day and the ways it can impact us in ways we might not expect.

Read the full story at news.emory.edu >>

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Emory investigates how COVID-19 affects the... - SaportaReport

CAMBRIDGE, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced the addition of Nancy Vitale as an independent director to its Board of Directors, effective as of September 15, 2020. Ms. Vitale brings more than 25 years of experience to Voyagers Board, with deep expertise in human resources. She is a former Senior Vice President and Chief Human Resource Officer at Genentech, a member of the Roche Group.

We are thrilled to welcome Nancy to our Board, said Andre Turenne, President and CEO of Voyager. Nancys accomplished background in helping organizations thrive by focusing on a strong patient-centric culture and employee wellbeing will be enormously valuable as we continue to grow Voyager.

Since leaving Genentech, in 2019, Ms. Vitale co-founded and continues to co-manage Partners for Wellbeing, LLC., a boutique human resources consulting firm. Prior to her 13-year tenure at Genentech, Ms. Vitale held senior human resources roles at Procter & Gamble Company and CIGNA. Ms. Vitale earned a Bachelor of Business Administration from the University of Michigan and an MBA from the Goizueta Business School of Emory University.

Im delighted to join Voyagers Board of Directors, Ms. Vitale commented. The company is led by an outstanding team and Im highly compelled by Voyagers mission to deliver life-changing medicines for patients suffering from severe neurological diseases. I look forward to working with other members of the Board and contributing to this important mission.

Ms. Vitale will also serve as a member of the Boards Compensation Committee.

About Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, Friedreichs ataxia, and other severe neurological diseases. For more information onVoyager Therapeutics, please visit the companys website atwww.voyagertherapeutics.com or follow@VoyagerTxon Twitter andLinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities law. The use of words such as may, might, will, should, expect, plan, anticipate, believe, estimate, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding Ms. Vitales participation as a member of Voyagers Board of Directors and her ability to help Voyager grow and develop a strong patient-centric culture and focus on employee wellbeing are forward-looking. All forward-looking statements are based on assumptions by Voyagers management that, although Voyager believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected, including the ability of Ms. Vitale to quickly integrate onto the Voyager Board of Directors, to make contributions as a member of the Voyager Board of Directors and to contribute to Voyagers culture. These statements are also subject to a number of material risks and uncertainties that are described in Voyagers most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, as updated by its future filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investors:Paul CoxVP, Investor Relations857-201-3463pcox@vygr.com

Media: Sheryl Seapy W2Opure949-903-4750sseapy@purecommunications.com

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Voyager Therapeutics Appoints Nancy Vitale to its Board of Directors - GlobeNewswire