Background: PTC Therapeutics yesterday announced results from the phase 3 “ACT DMD” trial of ataluren, an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD). The trial results showed clinically meaningful benefits for ataluren-treated patients, indicating the drug may slow functional decline in DMD patients, with some patients apparently . . .
Antoine de Morrée, a postdoctoral scholar at Palo Alto Veterans Institute for Research and Stanford University in Palo Alto, Calif., is testing a way to stop muscles from making toxic DUX4 protein as a possible treatment forfacioscapulohumeral muscular dystrophy (FSHD). The goals of de Morrée’s work are to understand how cells make stabile RNA messages, . . .
Legislation Helps Ensure Access to Clinical Trials for People with Rare Diseases On October 7, 2015, President Obama signed into law the Ensuring Access to Clinical Trials Act of 2015 (S. 139/H.R. 209) (EACT), which removes a barrier to participating in rare disease clinical trials. EACT makes permanent a law that allows individuals to participate . . .
Background: Sarepta Therapeutics reported today additional long-term clinical efficacy and safety data from its pivotal phase 2b program of eteplirsen to treat Duchenne muscular dystrophy (DMD). The data demonstrated that over a three-year period of time, trial participants who were treated with eteplirsen experienced a slower rate of decline and were, on average, able to . . .
In September, the Data Safety and Monitoring Board (DSMB), constituted to oversee the Diaphragm Pacing System in Participants with Amyotrophic Lateral Sclerosis (DPS in ALS) study, being conducted in the United States, issued a recommendation that new enrollment be halted. At the same time, the DSMB recommended that patients already randomized to the diaphragm pacing . . .
