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Press Releases

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
CITGO Celebrates 30th Anniversary of National Partnership with MDA

Submitted by kschaaf on Wed, 06/01/2016 - 09:42
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RAFAEL, Calif., May 31, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee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.
Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date

Submitted by corphan on Wed, 05/25/2016 - 06:51

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 25, 2016--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has notified the Company that they are continuing their review and internal discussions related to our pending NDA for eteplirsen and will not be able to complete their work by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016.
Santhera Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne Muscular Dystrophy (DMD)

Submitted by corphan on Tue, 05/03/2016 - 09:36

Santhera Pharmaceuticals (SIX: SANN) announces that it has submitted comprehensive briefing material and a meeting request to the FDA to discuss the filing of a New Drug Application (NDA) for Raxone® (idebenone) for the treatment of DMD patients not taking concomitant glucocorticoids.

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Press Releases

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

CITGO Celebrates 30th Anniversary of National Partnership with MDA

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

Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date

Santhera Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne Muscular Dystrophy (DMD)

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About Us

Press Releases

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

CITGO Celebrates 30th Anniversary of National Partnership with MDA

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

Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date

Santhera Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne Muscular Dystrophy (DMD)

Find MDA in your Community

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in your Community