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We're here to transform the lives of people affected by neuromuscular disease.

Press Releases

  • Marathon Pharmaceuticals Announces Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD)

    Submitted by corphan on Thu, 03/10/2016 - 10:53

    Patients Can Now Receive Deflazacort, an Investigational Treatment from Marathon, at No Cost Through Medical Research Centers Across U.S.

    Full Press Release: http://www.prnewswire.com/news-releases/marathon-pharmaceuticals-announc...

  • MDA Shamrocks Help Fight Muscular Dystrophy

    Submitted by kjerome on Tue, 03/01/2016 - 08:18

    CHICAGO, March 1, 2016— Tens of thousands of retailers nationwide have rallied together to raise funds to help the Muscular Dystrophy Association (MDA) save and improve the lives of kids and adults with muscular dystrophy, ALS and related life-threatening diseases that severely weaken muscle strength and mobility by participating in the 34th annual MDA Shamrocks program —the nation’s largest St. Patrick's Day fundraising program of its kind.

  • FAQ – Eteplirsen for DMD

    Submitted by studiog on Fri, 02/12/2016 - 11:27

    The experimental drug eteplirsen, under development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD), is under review by the U.S. Food and Drug Administration (FDA) for market approval. It marks an exciting and historic time for the MDA community and an important milestone for DMD treatments. Before a final decision is rendered, however, an FDA Advisory Committee will review the drug and provide a recommendation to the regulatory agency.

  • FDA Tells PTC Therapeutics That Ataluren (Translarna) Is Not Ready For Review At This Time

    Submitted by jcook on Tue, 02/23/2016 - 13:16

    Background: PTC Therapeutics today announced that it received a Refuse to File letter from the U.S. Food and Drug Administration (FDA) regarding PTC’s New Drug Application (NDA) for ataluren (brand name Translarna) to treat Duchenne muscular dystrophy (DMD).

    In the letter, the FDA states that the application was not sufficiently complete to permit a substantive review. PTC is reviewing the content of the letter to determine the appropriate next steps.

  • Lowe’s Supports MDA Shamrocks Program and Summer Camps to Help Families and Children with Muscular Dystrophy

    Submitted by studiog on Tue, 02/16/2016 - 14:49

    CHICAGO, Feb. 16, 2016 — Lowe’s stores nationwide unite to participate in the Muscular Dystrophy Association’s iconic Shamrocks program now through March 31. This year marks 15 years of Lowe’s Shamrocks support that helps improve lives in communities across the country and build stronger futures for kids and adults with muscular dystrophy, ALS and related life-threatening diseases that severely weaken muscle strength and mobility.

  • FDA Extends PDUFA Goal Date for Eteplirsen

    Submitted by kjerome on Tue, 03/22/2016 - 06:37

    Background: Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has extended the PDUFA goal date for eteplirsen by a standard extension period of three months. The new date by which the FDA must make a decision about whether to approve eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) is May 26, 2016.

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