Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.
PTC is moving forward with ataluren
PTC Therapeutics' phase 3 trial of its experimental drug ataluren remains open to boys with Duchenne or Becker MD caused by premature stop codon mutations
PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, wants to remind families affected by Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by specific genetic mutations that its phase 3 trial of
PTC Therapeutics CEO Stuart Peltz discusses next steps for ataluren, an experimental 'stop codon read-through' drug for Duchenne and Becker MD caused by specific mutations
Trials of the experimental muscular dystrophy drug ataluren showed that the drug was generally well-tolerated and slowed the rate of decline in walking ability, compared to the placebo group.
After consulting with regulatory agencies in Europe and the United States, biopharmaceutical company PTC Therapeutics has decided to move ahead with a global, phase 3 confirmatory study of ataluren. Trial...
After restructuring its agreement with Genzyme, PTC will continue developing ataluren for nonsense-mutation DMD/BMD and will study the drug in former trial participants
Update (July 25, 2012): This story was updated to reflect the fact that PTC has announced its intention to begin an open-label trial of ataluren in DMD/BMD for former ataluren trial participants in Europe, Israel and Australia.
PTC Therapeutics, with support from MDA, is testing ataluren in boys with DMD or BMD who have nonsense mutations and are no longer walking.
Update (March 3, 2010): An update to the article Ataluren Results Disappointing was posted.
Featured in this issue: Duchenne and Becker MD ** Facioscapulohumeral MD ** Friedreich's ataxia ** Limb-Girdle muscular dystrophy ** Myotonic dystrophy ** Spinal muscular atrophy