PTC Therapeutics' large-scale multinational trial of ataluren for nonsense-mutation Duchenne or Becker MD has opened its first site in Cincinnati, Ohio
A large-scale, multinational, phase 3 trial of the experimental drug ataluren has opened its first trial site, in Cincinnati, Ohio.
In early 2011, clinical trials of ACE-031, a myostatin inhibitor, were halted pending resolution of safety issues; in May 2013, the developers discontinued work on the drug
ARMGO Pharma is developing a novel drug based on a new strategy for treating muscle weakness and degeneration in Duchenne MD, Becker MD and potentially other muscle disorders
MDA has awarded $1 million to biopharmaceutical company ARMGO Pharma for development of a new strategy for treating Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).
FDA has asked Sarepta Therapeutics for additional information about dystrophin production and safety before it will consider accepting an application for accelerated approval of its exon-skipping drug
The U.S. Food and Drug Administration (FDA) has said it will consider accepting an application for accelerated approval for eteplirsen, an experimental drug for Duchenne muscular dystrophy (DMD), after it reviews additional data from clinical trials of the drug.
Weekly injections of the exon-skipping drug drisapersen in boys with Duchenne muscular dystrophy resulted in a significant difference in walking distance compared to a placebo
10 boys with Duchenne muscular dystrophy continue to show stabilization of walking ability; further trial results will be announced at an MDA conference in late April
Eteplirsen, an experimental exon-skipping therapy designed to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show sustained benefit on walking distance through week 74 of a phase 2b, 12-person study.
MDA-supported researchers have developed a research mouse with the same molecular abnormalities that underlie human types 1 and 2 facioscapulohumeral muscular dystrophy
A newly developed research mouse that has the same combination of genetic alterations that causes human facioscapulohumeral muscular dystrophy (FSHD) is expected to change the way research in this disease is conducted, possibly speeding the development of therapies.
Unlike humans, mice normally do not have a DNA structure called a D4Z4 repeat array that, when altered, causes FSHD. Therefore, FSHD...