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Press Releases

America’s Fire Fighters Fill the Boot to Support Kids and Adults with Muscular Dystrophy, ALS and Related Diseases

Submitted by kschaaf on Thu, 07/28/2016 - 11:46

Annual Fill the Boot drives heat up this summer to help bring strength, independence and life to those affected
FDA Turns Down Santhera Pharmaceuticals’ Request for Accelerated Approval for Idebenone to Treat DMD

Submitted by jcook on Fri, 07/22/2016 - 10:57

Background: Santhera Pharmaceuticals has been notified by the U.S. Food and Drug Administration (FDA) that the company must include results from its SIDEROS clinical trial at the time that it files a New Drug Application (NDA) for idebenone to treat Duchenne muscular dystrophy (DMD).
MDA and CMTA Partner to Advance Treatments and Care for Charcot-Marie-Tooth Disease

Submitted by jcook on Mon, 07/11/2016 - 14:17

CHICAGO, July 12, 2016 – The Muscular Dystrophy Association (MDA) and the Charcot-Marie-Tooth Association (CMTA) today announced a partnership aimed at advancing Charcot-Marie-Tooth disease (CMT) research, therapy development and clinical care. The partnership also aims to increase awareness and understanding about the disease by improving education for kids and adults affected by CMT, medical professionals and the public.
American Academy of Neurology, American Brain Foundation and Muscular Dystrophy Association Offer New Research Award

Submitted by kschaaf on Mon, 08/22/2016 - 11:29

CHICAGO, July 7, 2016 - The American Academy of Neurology (AAN), the American Brain Foundation and the Muscular Dystrophy Association (MDA) have announced a new Clinical Research Training Fellowship in muscular dystrophy for 2017.
Marathon Pharmaceuticals Announces Submission of Deflazacort New Drug Application to the FDA

Submitted by jcook on Tue, 06/14/2016 - 12:35

Drug Has Fast Track Status, Orphan Drug Designation and Rare Pediatric Disease Designation for Duchenne Muscular Dystrophy
BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa (drisapersen) in Europe

Submitted by jcook on Wed, 06/01/2016 - 11:09

SAN RAFAEL, Calif., May 31, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting. Those discussions clearly indicated that the CHMP intended to issue a negative opinion. Kyndrisa is an experimental drug for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

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About Us

Press Releases

America’s Fire Fighters Fill the Boot to Support Kids and Adults with Muscular Dystrophy, ALS and Related Diseases

FDA Turns Down Santhera Pharmaceuticals’ Request for Accelerated Approval for Idebenone to Treat DMD

MDA and CMTA Partner to Advance Treatments and Care for Charcot-Marie-Tooth Disease

American Academy of Neurology, American Brain Foundation and Muscular Dystrophy Association Offer New Research Award

Marathon Pharmaceuticals Announces Submission of Deflazacort New Drug Application to the FDA

BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa (drisapersen) in Europe

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About Us

Press Releases

America’s Fire Fighters Fill the Boot to Support Kids and Adults with Muscular Dystrophy, ALS and Related Diseases

FDA Turns Down Santhera Pharmaceuticals’ Request for Accelerated Approval for Idebenone to Treat DMD

MDA and CMTA Partner to Advance Treatments and Care for Charcot-Marie-Tooth Disease

American Academy of Neurology, American Brain Foundation and Muscular Dystrophy Association Offer New Research Award

Marathon Pharmaceuticals Announces Submission of Deflazacort New Drug Application to the FDA

BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa (drisapersen) in Europe

Find MDA in your Community

Find MDA
in your Community