An expression of the live unlimited spirit, stories and voices of the MDA community
The Muscular Dystrophy Association recently awarded an MDA human clinical trial grant for the development of a critical biomarker for Charcot-Marie-Tooth disease (CMT) to Mary M. Reilly, professor of clinical neurology and consultant neurologist, UCL Institute of Neurology and National Hospital for Neurology and Neurosurgery, London. The investment, totaling $1 million over three years, will . . .
After significant collaborative work among many stakeholders in the spinal muscular atrophy (SMA) community, we are pleased to share that the Secretary of Health and Human Services (HHS), Dr. Alex Azar, has accepted the recommendation to add SMA to the Recommended Uniform Screening Panel (RUSP) for newborns. This is a landmark decision for the SMA . . .
On March 29, fire fighters from departments throughout the northeast gathered at Mohegan Sun Casino in Connecticut for MDA’s annual Fill the Boot Camp event. The goal of the one-day gathering is to provide fire fighters with the training and information needed to have a successful Fill the Boot season and to celebrate the accomplishments . . .
Former NFL great Dwight Clark died from ALS (amyotrophic lateral sclerosis) on June 4, a little more than one year after revealing he had the disease. Clark was immortalized when he caught the winning touchdown pass in the NFC Championship Game in January 1982, sending the San Francisco 49ers to their first Super Bowl. The . . .
This morning, Summit Therapeutics announced that it is discontinuing development of ezutromid for Duchenne muscular dystrophy after primary and secondary endpoints were missed after 48 weeks of treatment in their PhaseOut DMD trial. PhaseOut DMD was a phase 2, multi-center, open-label clinical trial of ezutromid, a utrophin-modulation therapy. Thirty-eight boys enrolled in the trial completed the 48-week regimen. The . . .
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 . . .
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 . . .
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 . . .
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 . . .
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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