News

The U.S. Food and Drug Administration (FDA), which in September 2016 approved Serepta Therapeutics’ Duchenne muscular dystrophy (DMD) treatment Exondys 51 (eteplirsen), now finds itself the subject of a lawsuit aimed at forcing the FDA to turn over information about the approval process. Charles Seife, a journalism professor at New…

Healthcare costs for patients with Duchenne muscular dystrophy (DMD) are about 10 times those of healthy people, and increase as patients age, according to a new analysis of claims data. The figures, which may aid in healthcare planning and evaluating costs of emerging treatments, were — for the first time —…

The Stephen J. Wampler Foundation has reached an agreement to purchase a new home for Camp Wamp, an outdoor wilderness adventure camp for children with physical disabilities. Previously a Girl Scouts of Northern California camp, the larger, 129-acre wilderness facility is at Deer Lake in the high Sierras near Lake…

Deflazacort delays loss of ambulation (LOA) in Duchenne muscular dystrophy (DMD) patients by an average of 3.8 years more than standard of care corticosteroid treatments prednisone/prednisolone, post-hoc analyses of a Phase 3 clinical trial found. The new conclusions follow a reassessment of data obtained in the placebo group of the completed ACT DMD clinical trial (NCT01826487). The goal…

JAR of Hope, a foundation that funds Duchenne muscular dystrophy (DMD) research, is working to get legislation approved to make September 10-16 Duchenne Muscular Dystrophy Awareness Week in New York state. The foundation, along with New York City Assemblyman Michael Cusick, will ask New York Gov. Andrew M. Cuomo on…

Santhera Pharmaceuticals has updated the timeline for the ongoing assessment by the European Committee for Medicinal Products for Human Use (CHMP) of its Duchenne muscular dystrophy (DMD) drug, Raxone (idebenone). In June 2016, the European Medicines Agency’s (EMA) CHMP validated a marketing authorization application (MAA) for Raxone…

Positive results from the HOPE-Duchenne clinical trial support further clinical development of Capricor Therapeutics’ therapy candidate CAP-1002 for treatment of cardiac anomalies caused by Duchenne Muscular Dystrophy (DMD). The company expects to start enrolling patients for a placebo-controlled clinical trial of intravenous CAP-1002 in DMD in the second…

CureDuchenne has partnered with the Muscular Dystrophy Organization Nepal (MDON) to help deliver better care to Duchenne muscular dystrophy (DMD)  patients in that country, and education and support for their families. According to CureDuchenne, DMD affects over 300,000 children globally. An article, “Lived Experience of Mothers with Duchenne…

Levels of a protein called creatine kinase (CK) are a valuable biomarker for advanced Duchenne muscular dystrophy (DMD), but now researchers say it may also be a strong predictor of patients’ lung function. The study, “Correlation of Serum Creatine Kinase Level With Pulmonary Function in Duchenne Muscular Dystrophy,” was…

Summit Therapeutics has completed patient enrollment for PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid to treat Duchenne muscular dystrophy (DMD). PhaseOut DMD (NCT02858362) aims to provide proof-of-concept for ezutromid through measuring certain endpoints related to muscle structure, health and function. The trial could potentially provide…