premature stop codon

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

A strategy to repair DNA in cells taken from boys with Duchenne muscular dystrophy has resulted in production of dystrophin protein molecules

posted on June 11, 2013 - 2:05pm
Permanent repair of a faulty gene has long been a goal of researchers working to develop gene-based therapies. But many current gene modification strategies that have entered clinical trials have been based on temporary forms of gene correction — treatments that will need to be given frequently throughout a person's life.

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.

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

posted on February 21, 2013 - 4:31pm
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...

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

An MDA grant will help develop RTC13, a drug designed to cause dystrophin protein production in DMD-affected muscle fibers

posted on February 9, 2011 - 11:39am
A new MDA translational research grant for $476,465 over three years will allow Carmen Bertoni at the University of California, Los Angeles (UCLA) to develop RTC13, an experimental compound designed to treat Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) caused by a specific type of flaw in the gene for the muscle protein dystrophin.