News

MoveDMD (NCT02439216) is a three-part, ongoing clinical trial investigating an oral drug — edasalonexent (CAT-1004) — as a potential therapy in boys, ages 4 to 7, with Duchenne muscular dystrophy (DMD). The drug targets NF-kB, a protein activated in DMD and shown to promote the disease by driving inflammation, muscle degeneration,…

An investigational therapy to treat congenital myotonic dystrophy, AMO-02 (tideglusib), has been granted fast track designation by the U.S. Food and Drug Administration (FDA), AMO Pharma has announced. Myotonic dystrophy (DM) is the most common form of muscular dystrophy (MD). DM type 1 is the consequence of a…

A proposed U.S. database of children with complex medical problems, including muscular dystrophy (MD), has raised concern at a respected public policy institute. Researchers at the Houston institute contend that the database could be used to discriminate against the children and their families, in particular to deny them health insurance. Dr.

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…