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  • Sarepta Reports Positive Preliminary Findings in DMD Gene Therapy Trial June 21, 2018
    This week, Sarepta Therapeutics announced encouraging preliminary results from a phase 1/2a gene therapy trial designed to assess the investigational drug AAVrh74.MHCK7.micro-Dystrophin in boys with Duchenne muscular dystrophy (DMD). After 90 days in the trial, the first three participants all showed robust expression of micro-dystrophin — a shortened version of the protein that is absent […]
  • With MDA Support, Clinical Research Network is Expediting the Development of New Therapies for FSHD June 20, 2018
    The Muscular Dystrophy Association recently awarded an MDA clinical research network grant to develop and maintain a core facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial Research Network (CTRN). The investment, totaling $1.2 million over three years, supports seven medical centers that specialize in FSHD research and clinical care and is targeted to spur advances in FSHD […]
  • Finding His Calling: Doctor Living with FSHD Enters the Field of Neuromuscular Medicine June 19, 2018
    Growing up, Marc van de Rijn was always interested in medicine. After briefly considering a career working with computers, a sudden health challenge after his freshman year of college made him realize that instead of sitting behind a desk, he wanted to work with people and become a doctor. “After my freshman year, I suffered […]
  • From Graduation Onward: MDA National Ambassador Justin Moy Looks Ahead June 14, 2018
    Spring 2018 has been an exceptionally busy time for National Ambassador Justin Moy. After being named 2018 MDA National Ambassador in March, Justin traveled to Japan with members of his school (with his MDA Camp Counselor serving as his care attendant). He also had several artistic endeavors this spring, exhibiting his artwork at a local […]
  • Fishing For Muscular Dystrophy is Building Buzz – and Aiming for More June 13, 2018
    Whenever Paul Robertson and his Fishing for Muscular Dystrophy (FFMD) team pull into a truck stop, they know it might be a while before they are able to leave. Although there to pump fuel, the caravan of trucks and boats adorned with the FFMD and MDA logos are such a big hit with fellow motorists […]

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