Monthly Archives: March 2017

Complete Cpf1-Based CRISPR Genome Editing System Available – Labmate Online

Posted: March 2, 2017 at 1:52 pm

Integrated DNA Technologies (IDT) is the first genomics company to develop and bring to market a complete ribonucleoprotein-based Cpf1 CRISPR system. The Alt-R A.s. Cpf1 CRISPR System inherits the optimised, efficient, and cost-effective traits of IDTs innovative Cas9-based system while taking advantage of Cpf1s natural AT-rich target sequence preference and ability to make staggered cuts. In addition, IDT is launching an associated range of CRISPR support tools to expand experimental options and capabilities for molecular biology researchers. The new tools extend the ease-of-use and performance of IDTs Alt-R system through options for fluorescent visualisation, enhanced nuclease transfection, and genome editing detection. Together, the new expanded Alt-R range breaks barriers to wider target spaces not addressable by Cas9 systems alone, and provides a level of flexibility in experimental design not previously possible.

IDTs Alt-R System already overcomes the limitations of using sgRNAs in the ribonucleoprotein (RNP) complex by enhancing editing efficiency and lowering toxicity. Now, in developing a complementary Cpf1-based system, IDT has opened up options for targeting AT-rich sequences. The new system includes the Alt-R A.s. Cpf1 nuclease, containing two integrated nuclear localisation sites, which complexes as an RNP with a minimal 41-44 nucleotide Alt-R CRISPR-Cpf1 crRNA. The system requires no tracrRNA, reducing potential reagent costs and experimental complexity.

The expanded range of Alt-R support tools now includes the Alt-R CRISPR-Cas9 tracrRNA5 ATTO 550, and Alt-R Electroporation Enhancers for Cas9 and Cpf1. The former allows in vivo fluorescent visualisation of the RNP complex and FACS enrichment of transfected cells without affecting RNP functionality, while the enhancers improve the efficiency of transfection using the Nucleofector (Lonza) and Neon (Thermo) electroporation systems. These new tools enable researchers to improve the overall efficiency of their genome editing over traditional methods. In addition, the new Alt-R Genome Editing Detection Kit provides a fast, easy, and low-cost method for confirming editing events with a T7 Endonuclease I (T7EI)-based mismatch detection system.

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Complete Cpf1-Based CRISPR Genome Editing System Available - Labmate Online

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Life expectancy levels to hit 90 – The Irish World Newspaper – The Irish World Newspaper

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Life expectancy among humans is expected to surpass 90 a feat previously thought impossible by scientists for the first time, according to new research.

Evidence suggests that women born in South Korea in 2030 will have an average life expectancy of 90.8 years, while men could reach 84.1 years. People around the world can expect to live longer, while those in the UK who reach 65 in 2030 can look forward to an additional 20- 23 life years.

The research was carried out by scientists from Imperial College London in collaboration with the World Health Organisation (WHO). It examined those from high-income countries such as the US, Canada, the UK, Germany and Australia, as well as those from developing economies like Poland, Mexico and the Czech Republic.

Lead researcher Professor Majid Ezzati from the School of Public Health said: We repeatedly hear that improvements in human longevity are about to come to an end.

Many people used to believe that 90 years is the upper limit for life expectancy, but this research suggests we will break the 90- year-barrier.

I dont believe were anywhere near the upper limit of life expectancy if there even is one.

The UKs average life expectancy at birth for women will be 85.3 years in 2030, while the average life expectancy of a UK man will be 82.5 years. The team also predicted a 65- year-old man in the UK in 2030 could expect to live an additional 20.9 years, while a 65-year-old woman in the UK could expect 22.7 more years.

Men in Ireland, meanwhile, were among the highest placed when it came to new life expectancy figures. At 83.2 years, men born in 2030 had the fourth-highest life expectancy in Europe, while with 21.7 additional life years expected for 65-year-olds in that year, Irish men placed fifth in the world behind Canada, New Zealand, Australia and South Korea. The research showed that the gap between men and women in terms of life expectancy continues to narrow.

Men traditionally had unhealthier lifestyles, and so shorter life expediencies. They smoked and drank more, and had more road traffic accidents and homicides, Professor Ezzati said. However as lifestyles become more similar between men and women, so does their longevity.

Colin Mathers, co-author from the WHO, added that the overall increase was due to those over 65 living longer combined with the decline in premature deaths among those in their 40s and 50s. The authors agreed that the results mean that there is a responsibility for Governments around the world to make health and social care a priority in the future.

The fact that we will continue to live longer means we need to think about strengthening the health and social care systems to support an ageing population with multiple health needs, said Professor Ezzati.

This is the opposite of what is being done in the era of austerity. We also need to think about whether current pension systems will support us, or if we need to consider working into later life.

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Life expectancy levels to hit 90 - The Irish World Newspaper - The Irish World Newspaper

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Stress During Pregnancy Linked To Eczema In Children – Huffington Post Canada

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New U.K. research suggests that stress before conception can increase the risk of eczema in children.

Carried out by the Medical Research Council Lifecourse Epidemiology Unit, University of Southampton, the study is the first to link maternal stress before conception with the risk of atopic eczema in future children.

For the research the study assessed the stress levels of women recruited to the Southampton Women's Survey before they were pregnant.

Participants were asked to report how stressed they were in their daily lives with a sub-group also asked about their psychological wellbeing.

Around 3,000 babies were born to women taking part in the survey, who were then assessed for eczema at the ages of six months and 12 months.

The results showed that more than one in six women reported that stress affected their health 'quite a lot' or 'extremely' before falling pregnant, with the analysis showing that the infants of these mothers had a 20 per cent higher likelihood of developing atopic eczema at age 12 months when compared with the children of other participants.

The results also suggested that stress and low mood experienced closer to the time of conception may have an even greater impact on eczema risk.

The team believe the findings support the idea that eczema partly originates as a baby develops in the womb, with Dr Sarah El-Heis, the study's lead researcher explaining, "maternal stress can release certain hormones that can have an effect on the baby's immune response, leading to an increased risk in conditions like eczema."

The research, published in the journal Clinical and Experimental Allergy, could now reveal ways of reducing the risk of the skin condition.

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Stress During Pregnancy Linked To Eczema In Children - Huffington Post Canada

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Stress, low mood before pregnancy increases eczema risk in … – Hindustan Times

Posted: at 1:52 pm

New UK research suggests that stress before conception can increase the risk of eczema in children.

A first-of-its-kind study that links maternal stress before conception with the risk of atopic eczema in future children has been carried out by the Medical Research Council Lifecourse Epidemiology Unit, University of Southampton in the UK.

For the research, the study assessed the stress levels of women recruited to the Southampton Womens Survey before they were pregnant. Participants were asked to report how stressed they were in their daily lives with a sub-group also asked about their psychological wellbeing.

Around 3,000 babies were born to women taking part in the survey, who were then assessed for eczema at the ages of six months and 12 months.

The researchers say maternal stress can release certain hormones that can have an effect on the babys immune response. (Shutterstock)

The results showed that more than one in six women reported that stress affected their health quite a lot or extremely before falling pregnant, with the analysis showing that the infants of these mothers had a 20% higher likelihood of developing atopic eczema at age 12 months when compared with the children of other participants.

The results also suggested that stress and low mood experienced closer to the time of conception may have an even greater impact on eczema risk. The team believes the findings support the idea that eczema partly originates as a baby develops in the womb, with Dr Sarah El-Heis, the studys lead researcher explaining, maternal stress can release certain hormones that can have an effect on the babys immune response, leading to an increased risk in conditions like eczema.

The research, published in the journal Clinical and Experimental Allergy, could now reveal ways of reducing the risk of the skin condition.

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National Psoriasis Foundation Celebrates 50 Years of Patient Support, Advocacy, Research, Education and Outreach – Yahoo Finance

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PORTLAND, Ore., Feb. 28, 2017 /PRNewswire/ -- This year, the National Psoriasis Foundation (NPF) celebrates 50 years of patient support, advocacy, research funding, education and outreach to the more than 8 million individuals living with psoriasis and psoriatic arthritis. Originally formed in 1966 as The Psoriasis Society of Oregon, and officially becoming the National Psoriasis Foundation in 1967, its mission is focused on driving efforts to cure psoriatic disease and dramatically improving the lives of those affected.

Throughout its first 50 years, NPF has made significant contributions in advancing the research of life-changing treatments that have helped millions of people. From the tar-based procedures and topical creams to the revolutionary biologic treatments that exist today, NPF has always supported innovative approaches to treating psoriasis and psoriatic arthritis. While these achievements are to be celebrated, there is still much work to be done to improve access to treatments and, ultimately, to find a cure.

"We have only scratched the surface of what could be possible in treatingand ultimately curingpsoriasis and psoriatic arthritis. Advancements in psoriatic disease research are happening all the time, and NPF remains committed to funding this research while offering programs and services that support even more patients," said Dr. Colby Evans, M.D., National Psoriasis Foundation board chair. "I am honored to be associated with an organization that has always been at the forefront of driving efforts to find a cure for psoriasis and psoriatic arthritis."

NPF has achieved many milestones over the past 50 years, including securing for the first time ever, line-item funding in a federal budget for skin disease research, successfully lobbying the U.S. Food & Drug Administration for the approval of methotrexate for the treatment of severe psoriasis and opening the world's first support center providing free personalized assistance for people with psoriatic disease. Most recently, members of the NPF Medical Board issued the first paper in the United States that outlines psoriasis treatment targets and goals for individuals and health care providers to work on together to achieve clear skin and ultimately reduce the burden of the disease.

"NPF has been a pioneer in supporting research efforts that have led to the groundbreaking treatments used today," said Randy Beranek, president and CEO of the National Psoriasis Foundation. "During our 50th year, we are excited to recognize the people and milestones that have contributed so much to the psoriatic disease community, and remain committed to supporting research efforts towards finding a cure and dramatically improving the health outcomes of all affected by psoriasis and psoriatic arthritis."

In remaining true to its mission, the theme of the NPF 50th Year Celebration is "Driving Discovery, Creating Community." This allows the Foundation to celebrate the achievements of the past 50 years and to recognize the individuals and milestones that have made significant contributions to the psoriatic disease community. To recognize its 50th year, NPF will be holding events throughout the year, starting with a formal celebration on June 8, 2017, at the Plaza Hotel in New York City, and followed by the National Volunteer Conference and Research Symposium August 4 6, 2017 in Chicago, IL, as well as various TeamNPF events across the country.

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During this year of celebration, NPF is committed to raising $2 million to fund several promising areas of research and programing, specifically early career research. Supporting the NPF 50th year campaign will ensure that researchers studying psoriatic disease will have the resources needed to turn their questions and theories into a cure.

To learn more about the NPF 50th Year Celebration and how to get involved, visit: https://www.psoriasis.org/NPF50th.

Over the last 50 years, the National Psoriasis Foundation (NPF) has become the world's leading nonprofit patient advocacy organization fighting for individuals with psoriasis and psoriatic arthritis. NPF leads this fight by driving efforts for a cure and improving the lives of the more than 8 million Americans affected by this chronic disease. To date, NPF has funded more than $15 million in research grants and fellowships, and to commemorate 50 years, NPF plans to raise an additional $2 million for early scientific career research programs in 2017 alone. Each year, NPF strives to support, educate and advocate on behalf of more individuals living with or caring for someone with the disease than ever before. As part of that effort, NPF established the Patient Navigation Center to offer personalized assistance to everyone with psoriasis or psoriatic arthritis. Join our community today and help drive discovery and create community for all living with psoriatic disease.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/national-psoriasis-foundation-celebrates-50-years-of-patient-support-advocacy-research-education-and-outreach-300414766.html

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Lilly to Present New Data for Taltz (ixekizumab) in Psoriasis and Psoriatic Arthritis at the American Academy of … – Yahoo Finance

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INDIANAPOLIS , Feb. 28, 2017 /CNW/ --Eli Lilly and Company (LLY) will highlight new Phase 3 data evaluating Taltz (ixekizumab) for the treatment of moderate-to-severe plaque psoriasis and active psoriatic arthritis at the 75th annual meeting of the American Academy of Dermatology (AAD), which will take place March 3-7, 2017 , in Orlando, Fla.

Lilly will present 14 abstracts, including one oral late-breaker presentation highlighting head-to-head data on the efficacy and safety of Taltz compared to Stelara* (ustekinumab) for the treatment of moderate-to-severe plaque psoriasis at 24 weeks.

"As the world's largest dermatology meeting, the American Academy of Dermatology annual meeting is an opportunity to highlight new information and data to experts in the dermatologic disease field," said Dr. Lotus Mallbris, global brand development leader, Taltz, Eli Lilly and Company. "Nearly one year after the approval of Taltz in the U.S. and Europe , we are proud to bring new data further demonstrating the efficacy and safety of Taltz to the American Academy of Dermatology annual meeting."

Studies, as well as the times and locations of the data sessions, are highlighted below.

Oral Late-Breaker Presentation

Saturday, March 4

Poster Presentations

Sunday, March 5

Indications and UsageTaltz (ixekizumab) is indicated for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONSTaltz is contraindicated in patients with a previous serious hypersensitivity reaction, such as anaphylaxis, to ixekizumab or to any of the excipients.

WARNINGS AND PRECAUTIONSInfectionsTaltz may increase the risk of infection. The Taltz group had a higher rate of infections than the placebo group (27% vs. 23%). Serious infections have occurred. Instruct patients to seek medical advice if signs or symptoms of clinically important chronic or acute infection occur. If a serious infection develops, discontinue Taltz until the infection resolves.

Pre-Treatment Evaluation for TuberculosisEvaluate patients for tuberculosis (TB) infection prior to initiating treatment with Taltz. Do not administer to patients with active TB infection. Initiate treatment of latent TB prior to administering Taltz. Patients receiving Taltz should be monitored closely for signs and symptoms of active TB during and after treatment.

HypersensitivitySerious hypersensitivity reactions, including anaphylaxis, angioedema and urticaria, have been reported with Taltz. If a serious hypersensitivity reaction occurs, discontinue Taltz immediately and initiate appropriate therapy.

Inflammatory Bowel DiseaseCrohn's disease and ulcerative colitis, including exacerbations, occurred at a greater frequency in the Taltz group (Crohn's disease 0.1%, ulcerative colitis 0.2%) than in the placebo group (0%) during clinical trials. During Taltz treatment, monitor patients for onset or exacerbations of inflammatory bowel disease.

ImmunizationsPrior to initiating therapy with Taltz, consider completion of all age-appropriate immunizations according to current immunization guidelines. Live vaccines should not be given with Taltz.

ADVERSE REACTIONSMost common adverse reactions (>1%) associated with Taltz treatment are injection site reactions, upper respiratory tract infections, nausea, and tinea infections.

Please see accompanying Prescribing Information and Medication Guide. Please see Instructions for Use included with the device.

IX HCP ISI 18JAN2017

About Taltz (ixekizumab)Taltz (ixekizumab) is an IgG4 monoclonal antibody that selectively binds with interleukin 17A (IL-17A) cytokine and inhibits its interaction with the IL-17 receptor. IL-17A is a naturally occurring cytokine that is involved in normal inflammatory and immune responses. Taltz inhibits the release of pro-inflammatory cytokines and chemokines.

About Moderate-to-Severe Plaque PsoriasisPsoriasis is a chronic, immune disease that affects the skin.1 It occurs when the immune system sends out faulty signals that speed up the growth cycle of skin cells.Psoriasis affects approximately 125 million people worldwide, approximately 20 percent of whom have moderate-to-severe plaque psoriasis.1,[2] Psoriasis can occur on any part of the body and is associated with other serious health conditions, such as diabetes and heart disease.1 The most common form of psoriasis, plaque psoriasis, appears as raised, red patches covered with a silvery white buildup of dead skin cells.1

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AboutEli Lillyand CompanyLilly is a global healthcare leader that unites caring with discovery to make life better for people around the world. We were founded more than a century ago by a man committed to creating high-quality medicines that meet real needs, and today we remain true to that mission in all our work. Across the globe, Lilly employees work to discover and bring life-changing medicines to those who need them, improve the understanding and management of disease, and give back to communities through philanthropy and volunteerism. To learn more about Lilly, please visit us atwww.lilly.comandnewsroom.lilly.com/social-channels.

P-LLYThis press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about Taltz (ixekizumab) as a treatment for moderate-to-severe plaque psoriasis, and reflects Lilly's current belief. However, as with any pharmaceutical product, there are substantial risks and uncertainties in the process of development and commercialization. Among other things, there can be no guarantee that Taltz will receive additional regulatory approvals or be commercially successful. For further discussion of these and other risks and uncertainties, see Lilly's most recent Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission. Except as required by law, Lilly undertakes no duty to update forward-looking statements to reflect events after the date of this release.

_____________________________ 1 Psoriasis media kit. National Psoriasis Foundation website. https://www.psoriasis.org/sites/default/files/for-media/MediaKit.pdf. Accessed February 23, 2017 . 2 Psoriasis. American Academy of Dermatology website. https://www.aad.org/media-resources/stats-and-facts/conditions/psoriasis. Accessed February 23, 2017 .

Refer to: Jen Dial ; dial_jennifer_kay@lilly.com; 317-220-1172 (Lilly Bio-Medicines)

* Lilly USA , LLC 2017. All rights reserved. Taltz is a registered trademark owned or licensed by Eli Lilly and Company, its subsidiaries or affiliates. Stelara is a registered trademark owned or licensed by Janssen Pharmaceutical Companies of Johnson & Johnson, its subsidiaries or affiliates.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/lilly-to-present-new-data-for-taltz-ixekizumab-in-psoriasis-and-psoriatic-arthritis-at-the-american-academy-of-dermatology-aad-annual-meeting-300414834.html

SOURCE Eli Lilly and Company

To view this news release in HTML formatting, please use the following URL: http://www.newswire.ca/en/releases/archive/February2017/28/c5919.html

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Lilly to Present New Data for Taltz (ixekizumab) in Psoriasis and Psoriatic Arthritis at the American Academy of ... - Yahoo Finance

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‘Psoriasis likely to recur if patients have anxiety, depression’ | The … – The Siasat Daily

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New Delhi: Psoriasis, a skin condition in which skin cells build up and form scales and itchy, dry patches, has high chances of recurrence if the patient is subject to anxiety and depression, say doctors.

According to the doctors, stigma attached to the skin disease also contributes towards its recurrence. Nearly 84 per cent people suffering from moderate-to-severe psoriasis face discrimination and humiliation, with at least 40 per cent of them witnessing unwelcoming glares in public spaces.

Psoriasis has a significant impact on a patients quality of life. As long as there is a stigma attached to these conditions, patients will continue to have flare ups induced by emotional distress as anxiety and depression are major causes of psoriasis recurrences, Rajiv Sekhri, Consultant Dermatologist at Fortis hospital said on Wednesday.

The doctors also said that at least 18 per cent of the patients have been frequently asked whether their condition is communicable, pointing to a grave lack of awareness on the disease.

Psoriasis is a skin condition that changes the life cycle of skin cells. It causes cells to build up rapidly on the surface of the skin. The extra skin cells form thick, silver scales and itchy, dry, red patches that are sometimes painful. It is a persistent, long-lasting disease.

Stating that the psoriasis can affect personal lives and mental health, Sekhri said a survey conducted last year showed 36 per cent people admitted to feeling ashamed of their skin and the way they look.

Forty eight per cent of the patients interviewed shared that psoriasis has impacted their professional life and 30 per cent of them feel that psoriasis has impacted past or current relationships. These results reflect lack of hope and self-esteem, with 31 per cent of people stating that they do not believe clear or almost clear skin is an achievable goal for them, said Sekhri quoting the global survey of 8,300 people with psoriasis.

Sonal Soin, Medical Director and founder of city based Aayna Clinic said that awareness as well as acceptance is very low for psoriasis among patients as well as society.

Most of the people think it is contagious and avoid shaking hands or eating with patients suffering from the condition. Psoriasis is not contagious, one cannot pass the skin condition from one person to another, she said.

Creating awareness about psoriasis can help improve the quality of life of psoriasis patients, Soin added.

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Psoriasis Drugs Market Forecasts 2016-2026 – Yahoo Finance

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LONDON, Feb. 28, 2017 /PRNewswire/ --

Details Psoriasis Drugs Your Guide to R&D, Opportunities and Potential Revenues What does the future hold for treating psoriasis? Visiongain's new report gives you multilevel revenue forecasts from 2016, helping you stay ahead in data. There you see financial results, R&D trends, opportunities and potential revenues.

Download the full report: https://www.reportbuyer.com/product/4031803/

In our study you examine predicted sales to 2026 at overall world market, submarket, product and national level. You also assess technologies, competitive forces and expected products. That work's purpose is to help you avoid falling behind in knowledge, missing business or losing influence.

So read on now to explore that industry and discover what its future market could be worth. See how our investigation can benefit your work.

Forecasts from 2016 to 2026 and other analyses to help you harness opportunities Why miss data you need? Why struggle to find it? Instead let our analysis guide you through the psoriasis drug market's present and future. That understanding could bring you quicker, easier results, also benefiting your influence and reputation for business insight.

Besides revenue forecasting to 2026, our new work shows you recent results, growth rates and market shares. There you discover original analysis, seeing commercial outlooks and developments (R&D). In our 169 page study you gain 74 tables and 63 charts to benefit your research, analyses, plans, decisions and presentations.

The following sections outline what you get in our updated investigation.

Sales predictions for the world market and submarkets What is that industry's potential? Discover in our report overall world revenue to 2026 for psoriasis-treating medicines. First you see individual revenue forecasts to 2026 for three main submarkets at world level: - Biological drugs - Topical treatments - Small-molecule systemic therapies.

How will those segments' revenues expand? Which classes of treatment will generate most sales? There you assess prospects for revenue growth, seeing where you can gain, assessing how that industry will compete and develop.

You find revenue predictions by product, too, seeing from 2016 how they can succeed.

Revenue predictions for 13 products to 2026 discover what their futures hold How will individual drugs perform from 2016 to 2026 at world level? Our report forecasts revenues of established brands and medicines expected from 2016.

In our work you assess 13 drugs, including these brands: - Humira (adalimumab) - Stelara (ustekinumab) - Enbrel (etanercept) - Remicade (infliximab) - Otezla (apremilast)

There you discover how high sales can go, to 2026, finding drugs and years with highest predicted growth and revenues. You also examine trends and competition. You see what is happening, understanding challenges, progress, competitors and opportunities.

You find geographical revenue predictions too.

National markets where will highest revenues and growth occur? In developed and developing countries, opportunities in psoriasis treatment will occur from 2016. You see where and how pharma companies can develop and gain.

Our analyses show individual revenue forecasts to 2026 for these 11 national markets: - US and Japan - France, Germany, Italy, Spain and UK - Brazil, Russia, India and China.

There you find countries with highest revenues and potential sales growth. You assess that technology's future, seeing progress and finding what it means, including emerging trends for those skin disorder treatments.

Events affecting developers, producers and sellers issues shaping that industry Our report explains forces affecting that industry and market from 2016, including these influences: - Research and development, including monoclonal antibodies, IL-12, IL-17, IL-23, PDE4, TNF, JAK and T-cell inhibition, and histamine receptor antagonism - Prevalence of that skin disease, as well as issues for patients and healthcare providers assess treatment needs - Competition from generic drugs, biosimilars and new biologics - Companies specialising in dermatology and autoimmune disorders explore capabilities and emerging trends to treat psoriasis - Advances in drug delivery to benefit companies, patients and payers, including lotion, ointment and foam.

In our survey you explore political, economic, social and technological questions, assessing outlooks for business. You also examine that pharmaceutical segment's strengths, weaknesses, opportunities and threats.

That way you discover what the present and future hold, seeing what is possible for those drug developers, producers and marketers. Stay ahead in knowledge.

Companies leading that industry and our overall revenue prediction for 2020 From 2016, treatments for psoriasis hold potential for investments, medical advances and higher revenues. Our work predicts the overall world market for psoriasis medicines will reach $11.4bn in 2020, with further expansion to 2026.

See what is possible there the expected gains. Our report shows you what technologies and firms hold greatest potential. You explore activities of these companies, among others: - Johnson & Johnson - AbbVie - Amgen - Pfizer - Novartis - Celgene - LEO Pharma.

Our analysis shows you participants' results, capabilities, developments and outlooks. R&D in that industry is strong. From 2016 the psoriasis market holds many opportunities for revenue growth. Our work explains that potential, helping you stay ahead in knowledge.

Main ways Psoriasis Drugs Market Forecasts 2016-2026 helps your work In particular our new investigation gives you these advantages to benefit your research, analyses, decisions, proposals and presentations: - Revenues to 2026 at world level, with forecasting of 3 submarkets and 13 products assess prospects for investments and sales growth - Forecasts to 2026 for 11 national markets in the Americas, Europe and Asia investigate leading countries for revenues and expected sales - Forces driving and restraining that industry's expansion discover what influences revenue gain, finding trends, developments and opportunities - Prospects for established competitors and rising companies explore companies' portfolios, results, R&D and outlooks for future success.

Information found nowhere else: see how you can gain why miss out? Our investigation gives you independent analysis. There you gain business intelligence found only in our work, finding where opportunities for revenue expansion exist.

See in our work how you could benefit your research, analysis and decisions. Explore progress and possibilities. Also find how you could save time and benefit your authority on those drugs, helping your reputation for commercial insight.

Medicines for psoriasis get revenue predictions now, seeing what the future holds Our new report is for everyone investigating drugs for inflammation and skin disorders. In our survey you explore revenue forecasts to 2026, with discussions and other data. Avoid missing out in knowledge instead please get our report here now. Download the full report: https://www.reportbuyer.com/product/4031803/

About Reportbuyer Reportbuyer is a leading industry intelligence solution that provides all market research reports from top publishers http://www.reportbuyer.com

For more information: Sarah Smith Research Advisor at Reportbuyer.com Email: query@reportbuyer.com Tel: +44 208 816 85 48 Website: http://www.reportbuyer.com

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Teenager’s sickle cell reversed with world-first therapy – BBC News

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Sioux City Journal
Teenager's sickle cell reversed with world-first therapy
BBC News
But the study does show the potential power of gene therapy to transform the lives of people with sickle cell. "I think it's very significant, essential they've given him his life back," said Dr Deborah Gill from the gene medicine research group at the ...
Gene therapy lets a French teen dodge sickle cell diseaseSioux City Journal
Gene therapy product shows early promise in sickle cell diseaseHealio
Gene therapy relieves sickle cell in world firstThe Borneo Post

all 8 news articles »

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Teenager's sickle cell reversed with world-first therapy - BBC News

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Gene Therapy: A Breakthrough for Sickle Cell Anemia? – Montana Standard

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WEDNESDAY, March 1, 2017 (HealthDay News) -- Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow -- causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant.

In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells -- which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible -- and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team, led by Dr. Marina Cavazzana, of Necker Children's Hospital's biotherapy department, focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait -- that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin -- to replace the abnormal "adult" hemoglobin seen in sickle cell.

The hope, Williams said, is that gene therapy will ultimately offer a one-time treatment that cures sickle cell. But no one knows yet whether that will happen.

According to Williams, two key questions are: What's the long-term safety? And will the altered stem cells last for a patient's lifetime?

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published March 1 in the New England Journal of Medicine.

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Gene Therapy: A Breakthrough for Sickle Cell Anemia? - Montana Standard

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