Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy (Sarepta Therapeutics Inc)

Edit Public Technologies 05 May 2016
DMD is a rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death ... In recent years, the 6MWT has been adapted to evaluate functional capacity in neuromuscular diseases and has served as the basis for regulatory approval of a number of drugs for rare diseases, with mean changes in the 6MWT ranging from 28 to 44 meters....

Acceleron Pharma Reports First Quarter 2016 Financial and Operational Results (Acceleron Pharma Inc)

Edit Public Technologies 05 May 2016
(Source. Acceleron Pharma Inc). May 5, 2016 ... Musculoskeletal Diseases ACE-083 advancing toward the initiation of a Phase 2 trial in facioscapulohumeral muscular dystrophy (FSHD), a neuromuscular disorder, in the second half of 2016 ... Acceleron is also advancing clinical programs in the fields of oncology and neuromuscular diseases and has a comprehensive preclinical research effort targeting fibrotic and other serious diseases ... 2016 2015....

WAVE Life Sciences Enters Collaboration with Pfizer to Develop Genetically Targeted Therapies for the Treatment ...

Edit Stockhouse 05 May 2016
WAVE Life Sciences Enters Collaboration with Pfizer to Develop Genetically Targeted Therapies for the Treatment of Metabolic Diseases ... WAVE Life Sciences Ltd ... (NYSE ... “This alliance is consistent with WAVE’s strategy to build and advance a portfolio of medicines for neurological and neuromuscular diseases, while working with partners with deep expertise in other important therapeutic areas ... About WAVE Life Sciences ... Media. ... or....

WAVE Life Sciences Enters Collaboration with Pfizer to Develop Genetically Targeted Therapies for the Treatment of Metabolic Diseases (Wave Life Sciences Ltd)

Edit Public Technologies 05 May 2016
(Source. Wave Life Sciences Ltd) ... CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 5, 2016-- WAVE Life Sciences Ltd ... (NYSE ... 'This alliance is consistent with WAVE's strategy to build and advance a portfolio of medicines for neurological and neuromuscular diseases, while working with partners with deep expertise in other important therapeutic areas ... About WAVE Life Sciences ... Forward Looking Information ... View source version on businesswire.com ... Source....

How Far Away is the Technology of Ready Player One?

Edit Kotaku 05 May 2016
Thanks to its endless references to 1980s video games, Ernest Cline’s 2011 book Ready Player One is hard not to like if you’re a gamer of a certain age. It makes for a rip-roaring read ... And it’s soon to be a film, too ... My well-thumbed copy of Ready Player One ... It provides feedback with neuromuscular electrical stimulation at up to 52 points across the body - and it’s made of neoprene, so you can definitely tell you’re in the future ... ....

Faculty awards honor clinical, teaching, research excellence May 5, 2016 (Vanderbilt University)

Edit Public Technologies 05 May 2016
(Source. Vanderbilt University) ... Jeff Balser, M.D., Ph.D., speaks at Tuesday's Spring Faculty Meeting in Light Hall ... Other projects include the study of how mutations alter the structure and function of an ion channel protein associated with long QT syndrome, a potentially fatal cardiac arrhythmia, and how the mutation-prompted misfolding of peripheral, myelin protein 22 results in the neuromuscular disorder Charcot-Marie-Tooth Disease....

Flex Pharma Reports First Quarter 2016 Financial Results

Edit Business Wire 04 May 2016
BOSTON--(BUSINESS WIRE)--Click to Tweet Flex Pharma, Inc. (NASDAQ. FLKS), a biotechnology company developing innovative and proprietary treatments for nocturnal leg cramps (NLC), spasms associated with severe neuromuscular conditions such as MS and ALS, and exercise-associated muscle cramps, today reported financial results for the quarter ended March 31, 2016 and provided an update on its clinical development and corporate activities....

Ionis Reports Financial Results and Highlights for First Quarter 2016 (Isis Pharmaceuticals Inc)

Edit Public Technologies 04 May 2016
(Source. Isis Pharmaceuticals Inc). CARLSBAD, Calif., May 4, 2016 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq. IONS) today reported its financial results for the first quarter were in line with the company's expectations. '2016 is off to a strong start ... Lynne Parshall, chief operating officer of Ionis Pharmaceuticals ... Parshall ... Parshall ... Hougen ... Data showing increases in neuromuscular electrophysiology measurements were also reported....

May 03, 2016: Santhera Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne Muscular Dystrophy (DMD) (Santhera Pharmaceuticals Holding AG)

Edit Public Technologies 03 May 2016
Importantly, the package also includes data from the Cooperative International Neuromuscular Research Group's (CINRG) Duchenne natural history study (DNHS), which, in collaboration with CINRG, were used to conduct the first prospectively planned external control group study to compare outcomes for patients participating in DELOS with matched, contemporaneously-observed patients from the CINRG DNHS....

Biogen Announces Intent to Spin off Its Hemophilia Business

Edit Stockhouse 03 May 2016
Biogen Announces Intent to Spin off Its Hemophilia Business. New Company Will Build upon Strength of Current Therapies for Hemophilia A and B. Biogen to Focus on Novel Therapies for Neurology ... “Biogen is poised to make a tremendous difference in the lives of millions of people suffering from diseases with a neuronal etiology, including neurodegeneration, neuromuscular disorders, neuropathic pain, and ophthalmological indications ... John G....

Biogen Announces Intent to Spin off Its Hemophilia Business (Biogen Idec Inc)

Edit Public Technologies 03 May 2016
(Source. Biogen Idec Inc). CAMBRIDGE, Mass.--(BUSINESS WIRE)--Biogen Inc. (NASDAQ. BIIB) today announced that it intends to spin off its hemophilia business as an independent, publicly traded company ... 'Biogen is poised to make a tremendous difference in the lives of millions of people suffering from diseases with a neuronal etiology, including neurodegeneration, neuromuscular disorders, neuropathic pain, and ophthalmological indications....

America’s Top 10 Youth Volunteers of 2016 Named at 21st Annual Prudential Spirit of Community Awards

Edit Stockhouse 02 May 2016
America’s Top 10 Youth Volunteers of 2016 Named at 21st Annual Prudential Spirit of Community Awards ... Mellon Auditorium, range in age from 10 to 18 ... Jungin Angie Lee, 17, of Naperville, Ill., a junior at Metea Valley High School, co-founded a nonprofit organization that has generated nearly $200,000 over the past nine years through annual fundraising events to help find a cure for her rare neuromuscular disease ... About NASSP ... Editors ... or....

Clare Szalkowski of Dubuque, Iowa Named One of America’s Top 10 Youth Volunteers of 2016

Edit Yahoo Daily News 02 May 2016
WASHINGTON--(BUSINESS WIRE)-- ... Mellon Auditorium ... This Smart News Release features multimedia. View the full release here ... “This ‘friend’ made fun of her,” said Clare ... Jungin Angie Lee, 17, of Naperville, Ill., a junior at Metea Valley High School, co-founded a nonprofit organization that has generated nearly $200,000 over the past nine years through annual fundraising events to help find a cure for her rare neuromuscular disease ... Editors....
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