A research study of the use of the CoughAssist machine is open to children and teens with Ullrich congenital muscular dystrophy or Bethlem myopathy
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
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
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
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
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
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,...
Eteplirsen-treated boys with DMD who could perform the six-minute walk test showed continued stability in walking distance at week 120 of a phase 2b trial
Update (Jan. 16, 2014): Read Eteplirsen Data Still Strong: Treated Boys with DMD Stable at 120 Weeks for a recap of Sarepta's Jan. 15 presentation.