Muscular Dystrophies

A phase 2 trial of Duchenne MD drug drisapersen found the high-dose group walked farther than the placebo group at 24 and 48 weeks

posted on March 18, 2014 - 4:10pm
Update March 27, 2014: Prosensa and United Parent Projects Muscular Dystrophy jointly presented an educational webinar for patients and families affected by Duchenne muscular dystrophy on March 25, 2014. The approximately one-hour presentation is archived at Events & Presentations on the Prosensa site and includes a thorough discussion of Prosensa's plans for drisapersen and other exon-...

An MDA-supported team has developed mice with transplanted muscle tissue from people with facioscapulohumeral MD that is likely to speed research  

posted on February 14, 2014 - 1:44pm
Researchers funded in part by MDA have created a new research mouse (mouse "model") that they say is likely to speed investigations in facioscapulohumeral muscular dystrophy (FSHD) and therapy development for this disease. It's expected to complement an existing FSHD mouse model.

A research study of the use of the CoughAssist machine is open to children and teens with Ullrich congenital muscular dystrophy or Bethlem myopathy

posted on February 14, 2014 - 11:59am
Children and adolescents with Ullrich congenital muscular dystrophy or Bethlem myopathy are invited to participate in a study of a daily breathing treatment regimen using a brand-name device called a CoughAssist.

Ataluren (PTC124) for Duchenne-Becker MD related to premature stop codons did not receive approval in Europe prior to completion of an ongoing phase 3 trial

posted on January 31, 2014 - 10:15am
The experimental drug ataluren, in development for Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by specific mutations in the gene for the dystrophin protein, will not receive conditional approval at this time from the European Medicines Agency (EMA). Ataluren's developer is requesting that the EMA re-examine the data.

MDA-supported researchers have developed compounds that block harmful interactions between a needed protein and abnormal RNA in cells with a type 2 myotonic dystrophy defect

posted on January 23, 2014 - 2:49pm
MDA-supported investigators have designed two new compounds that precisely target the molecular defect that underlies type 2 myotonic muscular dystrophy (MMD2, or DM2), based on atomic-level imaging studies of the defect, also conducted in their laboratory.

Dutch biopharmaceutical company Prosensa outlined plans for drisapersen and other drugs for Duchenne MD in a Jan. 16, 2014, conference presentation and press release

posted on January 17, 2014 - 3:53pm
Update (Jan. 21, 2014): Both the Jan. 16, 2014, webcast from the J.P. Morgan conference and the Jan. 21 webcast for patient groups are available on the Prosensa site at Events & Presentations. The patient group webcast recaps many of the same themes as those summarized below from the J.P. Morgan conference presentation.

Sarepta Therapeutics presented new favorable safety and efficacy results for its experimental exon-skipping drug eteplirsen, designed to treat Duchenne MD caused by specific dystrophin mutations

posted on January 16, 2014 - 3:48pm
Update (Feb. 5, 2014): An analysis of pulmonary function tests at 120 weeks (2.3 years) found boys treated with eteplirsen showed a stabilization of respiratory function over a time period and in an age group where a decline would have been expected. Sarepta announced these results in a Feb. 5, 2014, press release.

Drisapersen developer Prosensa will update the investor community via Jan. 16 webcast and the DMD patient community via Jan. 21 webcast

posted on January 16, 2014 - 11:58am
Dutch biopharmaceutical company Prosensa, developer of the experimental drug drisapersen and other experimental compounds for Duchenne muscular dystrophy (DMD), will update the investor community via a Thursday, Jan. 16, 2014, presentation at the J.P. Morgan Healthcare Conference in San Francisco. The presentation will be webcast at 10 a.m. Pacific time/1 p.m. Eastern time. On Tuesday, Jan. 21,...