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  • Marathon Pharmaceuticals Submits New Drug Application to FDA for Deflazacort to Treat DMD June 14, 2016
    Marathon Pharmaceuticals announced today it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug deflazacort for the treatment of people with Duchenne muscular dystrophy (DMD). The FDA has 60 days to determine whether the NDA is complete and acceptable for filing. Deflazacort, a glucocorticoid, works […]
  • Boston Fire IAFF Local 718 & MDA Dedicate Statue in Honor of More than 60 Years of Partnership in the Fight Against Muscular Dystrophy June 12, 2016
    More than 100 Boston fire fighters and dignitaries, along with the the International Association of Fire Fighters (IAFF),  MDA and Boston Fire IAFF Local 718, united today on Memorial Sunday, an annual day to honor Boston’s fallen fire fighters, to celebrate more than 60 years of partnership in the fight against muscular dystrophy, ALS and […]
  • FDA Has Requested Additional Data for Eteplirsen Review June 7, 2016
    Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has requested that the company provide dystrophin data from biopsies that were taken as part of the ongoing confirmatory study of eteplirsen, called PROMOVI. The data was requested by the agency in connection with its ongoing evaluation of Sarepta’s New Drug Application (NDA) […]
  • Family Meeting Will Bring Together Individuals with RYR1-Related Muscle Weakness June 6, 2016
    Individuals with central core disease (CCD), centronuclear myopathy (CNM), multiminicore myopathy (MMC) or other diseases caused by a mutation in the ryanodine receptor (RYR-1) are invited to attend the first-ever RYR-1 International Family Conference, organized by the RYR-1 Foundation, July 22-24, 2016, in Baltimore. At the conference, you will be able to view presentations by […]
  • Announcing MDA Amplify June 6, 2016
    MDA Amplify is a new mobile app that revolutionizes the way you — an MDA supporter, partner or family — can share MDA news on your own social media channels. To become an MDA Amplifier, download the MDA Amplify app from iTunes or Google Play store (it’s free!), connect your personal social media accounts, and […]

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