nonsense mutation

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

posted on November 5, 2014 - 4:03pm
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

posted on December 9, 2013 - 9:37am
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' large-scale multinational trial of ataluren for nonsense-mutation Duchenne or Becker MD has opened its first site in Cincinnati, Ohio

posted on May 8, 2013 - 10:31am
Update (Oct. 31, 2013): This story has been updated to reflect that several trial sites are open to new participants. A large-scale, multinational, phase 3 trial of the experimental drug ataluren has opened at several sites.

New drug trials in Becker and Duchenne muscular dystrophies are open, and a new strategy to improve Pompe disease treatment is proposed

posted on June 28, 2012 - 2:11pm
PTC begins non-US study of ataluren in DMD/BMD

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

posted on September 15, 2011 - 9:24am
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.

The drug gentamicin increased dystrophin levels in boys with DMD due to a nonsense mutation, but didn't improve strength at the doses used

posted on April 14, 2010 - 3:34am
Results of the MDA-supported trial of gentamicin in Duchenne muscular dystrophy (DMD) were presented Wednesday, April 14, at the annual meeting of the American Academy of Neurology (AAN), held in Toronto. Levels of the needed dystrophin protein increased in six out of 12 participants who received the drug for six months. No functional improvements were seen.