People with HIV, particularly patients in sub-Saharan Africa and Latin America, have a high burden of HIV-associated neurocognitive disorder (HAND). This indicates the need for earlier neurologic care and initiation of antiretroviral therapy in this population, according to study results published in Neurology.

HIV, being a neurotropic virus, can invade the central nervous system during early infection, which is often associated with neurological complications like HAND. Study researchers sought to evaluate the burden and prevalence of HAND, as well as related factors in the global community living with the HIV.

This study was a meta-analysis of data from 123 cross-sectional and cohort studies that reported the prevalence of HAND in 35513 adults with HIV from 32 different countries. The pooled prevalence of HAND was estimated, and the overall worldwide burden of the disorder was evaluated.

Across studies, the diagnostic criteria for HAND consisted of the 2007 Frascati criteria in 64 studies, Global Deficit Score in 25 studies, and mental status examinations in 34 studies. Overall, the estimated prevalence of HAND in adults living with HIV was 42.6% (95% CI, 39.7-45.5). The prevalence of asymptomatic neurocognitive impairment (ANI) was 23.5% (95% CI, 20.3-26.8). Additionally, the prevalence of mild neurocognitive disorder (MND) was 13.3% (95% CI, 10.6-16.3) and of HIV-associated dementia (HAD) was 5.0% (95% CI, 3.5-6.8).

The prevalence of HAND was lower in individuals with a high level of nadir CD4 count (mean/median CD4 nadir <200, 45.2%; 95% CI, 40.5-49.9) compared with individuals with a low level of nadir CD4 count (mean/median CD4 nadir 200, 37.1%; 95% CI, 32.7-41.7).

The estimated worldwide number of patients with HIV and HAND was approximately 16,145,400 (95% CI, 15,046,300-17,244,500). The majority of cases were in sub-Saharan Africa, which was estimated to be in 72% of this population (n=11,571,200; 95% CI, 9,600,000-13,568,000).

Limitations of this meta-analysis were the inclusion of studies with varying definitions and diagnostic criteria for HAND and the use of screening tools that were limited when applied to certain target populations. Additionally, the use of Frascati criteria and the inclusion of patients with comorbidities that potentially contribute to cognitive impairment may have led to an overestimation of HAND prevalence in adults with HIV.

Based on the findings, the study researchers concluded that HAND should be prioritised among policy makers and HIV health-care providers for improved detection and efficient management of HAND integrated into routine clinical care in people with HIV.

Reference

Wang Y, Liu M, Lu Q, et al. Global prevalence and burden of HIV-associated neurocognitive disorder: a meta-analysis. Published online September 4, 2020. Neurology. doi:10.1212/WNL.0000000000010752

Read more here:
People With HIV Have High Burden of HIV-Associated Neurocognitive Disorder - Neurology Advisor

NEW YORK, Oct. 12, 2020 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc.(NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that four abstracts from the OV101 (gaboxadol) clinical development program will be presented at the Child Neurology Society/International Child Neurology Association (CNS/ICNA) 2020 Virtual Congress, taking place October 12 October 23, 2020.

The presentations include additional data and analyses from the Phase 2 ROCKET clinical trial of OV101 in individuals with Fragile X Syndrome; data on seizure and EEG outcomes from the Phase 2 STARS clinical trial in individuals with Angelman Syndrome; encore presentations of a study of caregiver insights in Angelman Syndrome; and the utility of the Clinical Global Impression (CGI) scale for studying outcomes in neurodevelopmental conditions.

Ovid is passionately committed to the development of OV101 to address the significant unmet medical need in individuals with both Angelman and Fragile X Syndromes two neurological conditions with tremendous caregiver and family impact with no approved treatment options for either, said Amit Rakhit, MD, MBA, President and Chief Medical Officer at Ovid. These data will contribute to evolving scientific exchange and advancing discussion around neurodevelopmental conditions, caregiver concerns, and the urgency to develop new medicines for these conditions.

Details of the presentations are as follows:

Title: Caregiver Insight on the Core Domains in Angelman Syndrome; Adera, et alPoster Number: #587

Title: The Phase 2a ROCKET Trial Investigating Gaboxadol (OV101) in Adolescents and Young Adults with Fragile X Syndrome; Berry-Kravis, et alPoster Number: #676

Title: The Adaptation and Utility of the Clinical Global Impression Scale for Studying Treatment Outcomes in Neurodevelopmental Conditions; Jaeger, et alPoster Number: #46

Title: STARS, a Phase 2 Safety, Tolerability, and Exploratory Efficacy Study of Gaboxadol in Adolescents and Adults with Angelman Syndrome: Seizure and EEG Outcomes; Wang, et alPoster Number: #588

AboutOvid TherapeuticsOvid Therapeutics Inc.is aNew York-based biopharmaceutical company using its BoldMedicineapproach to develop medicines that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of potential first-in-class medicines. The Companys most advanced investigational medicine, OV101 (gaboxadol), is currently in clinical development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935 (soticlestat) in collaboration with Takeda Pharmaceutical Company Limited for the potential treatment of rare developmental and epileptic encephalopathies (DEE). For more information on Ovid, please visitwww.ovidrx.com.

About OV101 (gaboxadol)OV101 is believed to be the only delta ()-selective GABAAreceptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has demonstrated in laboratory studies and animal models to selectively activate the -subunit of GABAAreceptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through modulation of tonic inhibition.

Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and thereby address several core symptoms of these conditions. In both these syndromes, the underlying pathophysiology includes disruption of tonic inhibition modulated through the -subunit of GABAAreceptors. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. This compound has also previously been tested in more than 4,000 patients (more than 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles. Ovid is conducting a pivotal Phase 3 clinical trial with OV101 in Angelman syndrome (NEPTUNE) and has completed a Phase 2 signal-finding clinical trial with OV101 in Fragile X syndrome (ROCKET).

OV101 has received Rare Pediatric Disease Designation from the FDA for the treatment of Angelman syndrome. The FDA has also granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. In addition, theEuropean Commission(EC) has granted orphan drug designation to OV101 for the treatment of Angelman syndrome. TheU.S. Patent and Trademark Officehas granted Ovid patents directed to methods of treating Angelman syndrome and Fragile X syndrome using OV101. The issued patents expire in 2035 without regulatory extensions.

Forward-Looking StatementsThis press release includes certain disclosures that contain forward-looking statements, including, without limitation, statements regarding the potential benefits, clinical and regulatory development, the likelihood that data will support future development, and the association of data with treatment outcomes. You can identify forward-looking statements because they contain words such as will, appears, believes and expects. Forward-looking statements are based on Ovids current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include uncertainties in the development and regulatory approval processes, and the fact that initial data from clinical trials may not be indicative, and are not guarantees, of the final results of the clinical trials and are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and/or more patient data become available. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovids filings with the Securities and Exchange Commission under the caption Risk Factors. Such risks may be amplified by the COVID-19 pandemic and its potential impact on Ovids business and the global economy. Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

Contacts

Investors and Media:Ovid Therapeutics Inc.Investor Relations & Public Relationsirpr@ovidrx.com

OR

Investors:Argot PartnersMaeve Conneighton212-600-1902ovid@argotpartners.com

Media:Argot PartnersJoshua R. Mansbach212-600-1902ovid@argotpartners.com

OR

Media:Dan Budwick1ABdan@1abmedia.com

See the original post:
Ovid Therapeutics to Present at the Child Neurology Society/International Child Neurology Association (CNS/ICNA) Virtual Congress - GlobeNewswire

October 13th, 2020 Baptist Health South Florida

Boca Raton, FL October 13, 2020 Khalid A. Hanafy, M.D., Ph.D., has joined Marcus Neuroscience Institute at Boca Raton Regional Hospital, part of Baptist Health, as medical director of neurocritical care and director of research. He specializes in the care of subarachnoid hemorrhage patients and the study of neuroinflammation. He also serves as associate professor of neurology at Florida Atlantic University Charles E. Schmidt College of Medicine in Boca Raton. He is board certified in neurology and neurocritical care.

Dr. Hanafy joined Marcus Neuroscience Institute from Beth Israel Deaconess Medical Center/Harvard Medical School in Boston, Mass., where he served as the director of the neurological intensive care unit and was an assistant professor of neurology at Harvard Medical School.

We are pleased to welcome Dr. Hanafy to Marcus Neuroscience Institute, said Frank D. Vrionis, M.D., MPH, Ph.D., Institute director and chief of neurosurgery. His clinical skills, research acumen and leadership in the field of neurology will greatly benefit our team and our patients.

As the Institutes director of research, Dr. Hanafy is principal investigator of cutting-edge studies that seek to bring the most advanced, personalized treatments to subarachnoid hemorrhage patients and improve their survival rates and health outcomes. His groundbreaking work in neuroinflammation has been funded by the National Institutes of Health, American Heart Association, American Academy of Neurology, and Massachusetts Institute of Technology. Marcus Neuroscience Institute is at the forefront of stem cell therapeutics, and Dr. Hanafy and Dr. Vrionis have already initiated clinical trials using stem cells in critically ill COVID patients. Together, they will expand research and clinical trials using stem cells for the treatment of other neurological conditions, such as stroke and brain tumors.

Dr. Hanafy has authored more than 40 articles, book chapters and invited editorials in peer-reviewed scientific publications and serves on the editorial boards of scholarly journals in his field. He is a member of several professional societies, including the American Academy of Neurology, Society for Neuroscience Research, Society of Critical Care Medicine, and Neurocritical Care Society.

Dr. Hanafy earned his medical degree and doctorate degree in molecular biology at the University of Texas Medical Scientist Training Program at Houston, a dual degree program of the University of Texas McGovern Medical School and MD Anderson Cancer Center UTHealth Graduate School of Biomedical Sciences. He did his thesis graduate work under Dr. Ferid Murad, the 1998 Nobel laureate. He returned to these Houston facilities to complete a neurology residency following an internal medicine internship at the University of Texas Southwestern at Parkland Memorial Hospital in Dallas. He concluded his medical training with a two-year fellowship in neurological critical care at Columbia University Medical Center in New York City.

About the Marcus Neuroscience InstituteThe Marcus Neuroscience Institute at Boca Raton Regional Hospital is an innovative nexus for neurologic and neurosurgical care. The 57,000-square-foot facility houses a 20-bed Neuro Intensive Care and Step-Down Unit, four dedicated operating rooms including one equipped with intraoperative MRI and two with intraoperative CT capability and a biplane angiography suite, a crucial component in the diagnosis and care of neurological conditions. The Institute has a staff of five neurosurgeons and nine neurologists who represent some of the most respected clinicians in their fields and is affiliated with Florida Atlantic Universitys Charles E. Schmidt College of Medicine.

About Boca Raton Regional HospitalBoca Raton Regional Hospital is part of Baptist Health South Florida, the largest healthcare organization in the region, with 11 hospitals, nearly 23,000 employees, more than 4,000 physicians and more than 100 outpatient centers, urgent care facilities and physician practices spanning across Miami-Dade, Monroe, Broward and Palm Beach counties. Baptist Health has internationally renowned centers of excellence in cancer, cardiovascular care, orthopedics and sports medicine, and neurosciences. In addition, it includes Baptist Health Medical Group; Baptist Health Quality Network; and Baptist Health Care On Demand, a virtual health platform. A not-for-profit organization supported by philanthropy and committed to our faith-based charitable mission of medical excellence, Baptist Health has been recognized by Fortune as one of the 100 Best Companies to Work For in America and by Ethisphere as one of the Worlds Most Ethical Companies. For more information, visit BaptistHealth.net/Newsroom and connect with us on Facebook, Instagram, Twitter and LinkedIn.

# # #

See the rest here:
Marcus Neuroscience Institute names Khalid A. Hanafy, M.D., Ph.D., Medical Director of Neurocritical Care and Research - Baptist Health South Florida

The Duke Neurology Departments Nada El Husseini, MD, and Andrew Spector, MD, both appeared in the American Academy of Neurologys (AAN) AAN Alumni Leadership Newsletter for October 2020, where they discussed transforming telestroke during a global health crisis and how neurologists can reduce racial health disparities.

El Husseini, a 2020 graduate of the AANs Transforming Leadership Program (TLP), spoke with Daniel Jose Correa, MD, MSC, about her year-long project to optimize telestroke care. El Husseini discussed her efforts to add standardized, patient-centered measures of outcomes in Dukes stroke network, improve wellness and satisfaction of telestroke providers, and include telestroke education in our Vascular Neurology Fellowship. Correa and El Husseini also discussed North Carolina and Dukes response to the COVID-19 pandemic, and how that pandemic affected (and continues to affect) telemedicine and the needs of patients with neurological conditions.

Spector, meanwhile, spoke with Ima Ebong, MD, as representatives of neurologists with different racial backgrounds to provide their perspectives on racial inequalities, and how the AAN and individual neurologists can make a difference.

Read both of these articles here in the AANs Leadership Alumni Newsletter.

See the original post:
El Husseini, Spector discuss transforming telestroke and racial disparities with AAN - Duke Department of Neurology

By: Explained Desk | New Delhi | Updated: October 14, 2020 7:24:43 pmResearchers also conclude that encephalopathy (a broad term used for diseases that alter brain structure and function) was found to be associated with increased mortality and morbidity. (Photo: AP)

Two recent studies have explored the neurological effects of COVID-19 on patients. While a research published in the journal Neurology has pointed out the various neurological manifestations, another published in the Annals of Clinical and Translational Neurology says that neurologic manifestations were present in nearly a third of the COVID-19 patients studied.

What does the new research tell us?

The first study, published in Neurology, was conducted across 11 hospitals and included 64 patients, out of which 43 were men and 21 were women. The median age of the patients was 66. Out of these, the MRIs of 36 patients (54 per cent) were considered abnormal and ischemic strokes, (27 per cent), leptomeningeal enhancement (17 per cent) and encephalitis (13 per cent) were the most common neuroimaging findings.

Confusion was the most common neurologic manifestation experienced by 53 per cent of the patients, followed by impaired consciousness (39 per cent), presence of clinical signs of corticospinal tract involvement (31 per cent), agitation (31 per cent) and headache (16 per cent).

Significantly, half of the patients considered for this study had acute respiratory distress syndrome (ARDS), while 11 per cent died.

Express Explainedis now onTelegram. Clickhere to join our channel (@ieexplained)and stay updated with the latest

The second study examined neurological manifestations in over 509 patients in a hospital network in Chicago, Illinois. This study found neurological manifestations in 215 patients (42.2 per cent) at the onset of the disease, in 319 patients (62.7 per cent) at hospitalisation, and at any time during the course of the disease in 419 patients (82.3 per cent).

As per the study, the most frequent neurologic manifestations were myalgias (44.8 per cent), headaches (37.7 per cent), encephalopathy (31.8 per cent), dizziness (29.7 per cent), dysgeusia (15.9 per cent), and anosmia (11.4 per cent). Further, the researchers found that strokes and movement disorders were uncommon among this cohort of patients, while 26.3 per cent of the patients required mechanical ventilation.

Significantly, researchers say that independent risk factors for developing neurological manifestations include severe COVID-19 and younger age.

Also in Explained | Should you get yourself tested for Covid-19 just to rule it out?

So what does this mean?

The findings from the first study imply that patients with COVID-19 can develop a wide range of neurologic manifestations, which may be associated with severe and fatal complications such as stroke and encephalitis.

On the other hand, the findings from the second study imply that neurologic manifestations occur in most hospitalised COVID-19 patients. Researchers also conclude that encephalopathy (a broad term used for diseases that alter brain structure and function) was found to be associated with increased mortality and morbidity, independent of disease severity.

As per a report in The New York Times, only 32 per cent of the patients with altered mental function in the second study were able to handle routine daily activities like cooking and paying bills.

The Indian Express is now on Telegram. Click here to join our channel (@indianexpress) and stay updated with the latest headlines

For all the latest Explained News, download Indian Express App.

The Indian Express (P) Ltd

Continued here:
Explained: Coronavirus impact on our brain what research says on types of damage, whos more vulnerable - The Indian Express

Testing someone for nerve damage is done manually by a clinician who determines what a patient can detect on their skin, most often using a specialized set of monofilaments or a tuning fork. It can be a long and uncomfortable process for the patient and the clinician. But using wearable technologies, Dr. Ryan Peters, PhD, in the Faculty of Kinesiology and member of the Hotchkiss Brain Institute at the Cumming School of Medicine, is developing new ways for performing neurological testing anytime, anywhere, and with greater accuracy.

Peters is a co-founder and chief science officer of Vibratus Inc., which is bringing these new neurological wearable technologies to market. You can think of Vibratus as your wearable neurologist, says Peters. He is pictured above, right, with members of his team.

Ryan Peters.

Adrian Shellard

Peters is creating a suite of devices and a software application, or app, to remotely monitor sensory and motor function of the foot sole, hand and other parts of the body. Vibratus technology uses vibration-emitting wearable devices, such as smartphones and watches, to measure a patients neural responses. This biometric data can be tracked and exchanged between the patient and their health-care provider at chosen intervals, enabling the worlds first virtual neurology clinic. The COVID-19 pandemic provides an ideal proving ground for such technologies, when in-person access to the neurologist can be more restrictive.

Patients with nerve damage have increased risk of injuries and falls, and yet months can go by before they can visit a neurologist to have their condition re-assessed. With this app, both the doctor and patient will quickly know if personalized treatment strategies are working or if they need to be adjusted, says Peters.

Peters says the technology is useful for nerve disorders such as peripheral neuropathy (nerve damage caused by conditions such as Type 2 diabetes or chemotherapy), peripheral nerve or spinal cord injuries, cerebral palsy, Parkinsons disease and stroke. The clinical tests that Vibratus is looking to improve upon are part of the standard neurological exam, making them useful for a broad range of neurological disorders.

Though still in the testing phase now, Peters plans to launch the app within the next year and has filed U.S. provisional patents for these technologies. It would be great to see our devices in the hands of everyone who does neurological testing. We have the potential to improve outcomes for people with neurological disorders while saving time and money for the health-care industry.

Vibratus will be the first startup business to come out of the Faculty of Kinesiologys new specialization in wearable technology, referred to as We-TRAC. Dr. Reed Ferber, PhD, who leads the We-TRAC program,says Peters has been a fantastic addition to the collaborative group.

He has shown success in obtaining grants, working with companies, and now as the first startup company to come out of We-TRAC, says Ferber. Peters says Ferber has been a tremendous mentor to him, and that it was Ferber who suggested he apply for the grants that are now supporting this endeavour.

As well as receiving a $101,500 grant from the National Sciences and Engineering Research Council of Canada, or NSERC, Peters received a $200,000 Life Science Fellowship from Innovate Calgary. These grants are for research and development funding, and the creation, entrepreneurship and talent development for emerging life science-based companies.

Innovate Calgary has supported our device prototyping activities and helped guide the business development for Vibratus which has been invaluable to us, says Peters.I appreciate the incredible support Im receiving from everyone.

Peters also collaborates closely with Drs. Tyler Cluff, Art Kuo and Nicole Culos-Reed in the Faculty of Kinesiology, along with his incoming students and the Vibratus team, includingco-foundersRaied Aburashed, who is the chief executive office and holds a Bachelor of Science degree from the Schulich School of Engineering at UCalgary, and Dr. Osman Darici, PhD, who is the chief technology officer and a postdoctoral scholar in the Faculty of Kinesiology at UCalgary.

For more information about the program, contact Dr. Ryan Peters, PhD.

We-TRACis developing the next generation of wearable tech experts and focusing on using wearable technology to revolutionize sport performance, health care and health research.

The University of Calgarys Faculty of Kinesiology is theNo. 1 ranked sport science school in North America and No. 7 globally, according to ShanghaiRanking.

Led by theHotchkiss Brain Institute,Brain and Mental Healthis one of six research strategies guiding the University of Calgary toward itsEyes Highgoals. The strategy provides a unifying direction for brain and mental health research at the university.

The University of Calgarys multidisciplinaryEngineering Solutions for Health: Biomedical Engineeringresearch strategy drives solutions to our most pressing health challenges in disease and injury prevention, diagnosis, and treatments. Our biomedical engineering researchers make a significant impact in our communities by extending lives, improving quality of life, promoting independence, and continuously improving the health system.

(Photos were taken prior to COVID-19)

Ryan Peters lab group in the Faculty of Kinesiology at the University of Calgary

Read the original post:
Kinesiology researcher creates smart way to assess and track neurological function - UCalgary News

The investment, which totals 492,535, will be directed specifically towards initiatives which focus on delivering treatment in partnership with the NHS and the third sector; and forms part of the governments 4.5m five-year action plan to ensure patients living anywhere in Scotland can access personalised neurological care and support.

The Neurological care and support: framework for action 2020-2025 programme sets out the governments vision for improving the quality of life of adult patients with neurological conditions by raising the standard and availability of care and treatment which they are able to access; as well as by sourcing and distributing information and support around living with neurological issues.

Joe FitzPatrick, Scotlands Minister for Public Health, said: Around one million people in Scotland live with a neurological condition ranging from common conditions such as migraines to life-limiting illnesses such as motor neurone disease (MND). We recognise the scale, variety and context of neurological conditions and how distressing they can be for the person with the condition and their family and the framework for action aims to support those responsible for providing care and support to people affected. This latest round of funding will ensure this vital work not only continues but is strengthened despite the challenges faced during the pandemic.

One recipient of the new funding is the PSP Association (PSPA), a charity which offers information and support to people affected by progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD). PSPA CEO Andrew Symons said: Neurological Care and Support Framework Funding will allow us to map provision for PSP and CBD in Scotland, simultaneously highlighting the information and support that we can offer from PSPA. This will contribute to the aim of earlier diagnosis of these rare conditions and reduce the time spent by people in the wrong part of the system with the result they can benefit from the provisions of the Framework as soon as their disease is recognised.

Recommended Related Articles

The rest is here:
Scotland allocates 500,000 for treatment of neurological conditions - Health Europa

CRANBURY, N.J., Oct. 12, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced additional positive interim results from its CLN6 Batten disease gene therapy program, AT-GTX-501. The results are featured in a virtual poster presentation at the Joint 16th International Child Neurology Congress and 49th Annual Child Neurology Society Meeting being held October 12-23, 2020. The presentation is also available in the Events and Presentations section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/events-and-presentations.

The Abigail Wexner Research Institute (AWRI) at Nationwide Childrens Hospital is conducting the ongoing Phase 1/2 clinical study of a single one-time intrathecal administration of AT-GTX-501 gene therapy for variant late-infantile neuronal ceroid lipofuscinosis 6 (vLINCL6) disease, also known as CLN6 Batten disease. With no approved treatments, CLN6 Batten disease is a fatal neurologic disease that rapidly robs children of their ability to walk, speak, think, and see.

Clinical Data Highlights:Interim safety data are available for 13 children with CLN6 Batten disease. Interim efficacy data are available for the first 12 children reaching the 12-month timepoint and for eight children up to 24 months, post-administration of the AAV-CLN6 gene therapy.

Jeff Castelli, Ph.D., Chief Development Officer ofAmicus Therapeutics, stated, We are pleased to share these positive interim clinical data for our intrathecal AAV gene therapy with the CLN6 community. The data continues to suggest that our gene therapy has the potential to be a treatment option for children living with CLN6 Batten disease, an ultra-rare, debilitating condition that leads to progressive declines in cognitive and motor function, and often results in death early in life.

Emily de los Reyes, M.D., Ph.D., Principal Investigator of the CLN6 clinical trial at AWRI at Nationwide Childrens andProfessor of Clinical Pediatrics and Neurology at The Ohio State University College of Medicine, stated, I remain pleased with the progress of this trial as well as our collection of natural history data to further inform the results for the AAV-CLN6 gene therapy. The interim results show that this investigational gene therapy has the potential to slow the neurological disease progression in children with CLN6 Batten disease.

Regulatory interactions for AT-GTX-501 are ongoing and the Company expects to provide feedback on the path forward in 2021.

Amicus has exclusive rights to the CLN6 gene therapy program developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About AT-GTX-501AT-GTX-501 is a novel gene therapy in Phase 1/2 development for CLN6 Batten disease, a rare, fatal, inherited lysosomal disorder with no approved treatment that primarily affects the nervous system. AT-GTX-501 is dosed in a one-time intrathecal infusion to deliver a functional copy of the CLN6 gene to cells of the central nervous system. The therapy is designed to address the underlying enzyme deficiency that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., AT-GTX-501 was granted Rare Pediatric Disease and Orphan Drug designations by the United States Food and Drug Administration. In the EU, the Company holds PRIME and orphan medicinal product designations.

About Batten DiseaseBatten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. In these disorders, a defect in a specific gene triggers a cascade of problems that interferes with a cells ability to recycle certain molecules. Each gene is called CLN (ceroid lipofuscinosis, neuronal) and given a different number designation as its subtype. There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. The various types of Batten disease have similar features and symptoms but vary in severity and age of onset.

Most forms of Batten disease/NCLs usually begin during childhood. The clinical course often involves progressive loss of independent adaptive skills such as mobility, feeding and communication. Patients may also experience vision loss, personality changes, behavioral problems, learning impairment and seizures. Patients typically experience progressive loss of motor function and eventually become wheelchair-bound, are then bedridden and die prematurely.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials and the prospects and timing of the potential regulatory approval of our product candidates. In particular, this press release relates to interim data from an ongoing Phase 1/2 study to investigate intrathecal administration of AAV-CLN6 gene therapy. The inclusion of forward-looking statements arising from this interim data, ongoing study and natural history preliminary data should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. The interim data and Phase 1/2 study discussed herein is inherently preliminary and early in the study, derived from a limited patient set, and later trial results with this patient set or others may not be consistent with these preliminary results. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2019 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2020. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.

CONTACTS:

Investors:Amicus TherapeuticsAndrew FaughnanDirector, Investor Relationsafaughnan@amicusrx.com(609) 662-3809

Media:Amicus TherapeuticsDiana MooreHead of Global Corporate Communicationsdmoore@amicusrx.com(609) 662-5079

FOLDG

Read this article:
Amicus Therapeutics Announces Additional Positive Interim Clinical Data for CLN6 Batten Disease Gene Therapy at 49th Annual Meeting of the Child...