A strategy to repair DNA in cells taken from boys with Duchenne muscular dystrophy has resulted in production of dystrophin protein molecules
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
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
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...
A study of the effects of two drugs on blood flow to exercising muscles needs four more boys with Duchenne MD in order to be complete
A 12-participant study of the acute effects of two vasodilating drugs on blood flow to exercising muscles needs four more boys with Duchenne muscular dystrophy (DMD) who meet study criteria and are able to travel to Los Angeles. Vasodilators increase the diameter of blood vessels.
GlaxoSmithKline's Duchenne MD exon-skipping drug moves into a phase 3 trial and is renamed drisapersen; "block skipping" of exons 45-55 in mice looks promising
Exon skipping is an experimental therapeutic strategy in which regions — exons — of a gene are targeted and blocked ("skipped") by laboratory-designed molecules. The goal is that the remaining genetic instructions will lead to production of a shorter but still-functional protein.
The experimental drug RTC13 caused dystrophin production and functional benefit in Duchenne MD research mice, outperforming similar drugs
An experimental drug called RTC13, designed to treat Duchenne muscular dystrophy (DMD) by restoring production of the muscle protein dystrophin, has shown promise in experiments in dystrophin-deficient mice that have a DMD-like disease.
RTC13's MDA-supported developers say they're optimistic about the compound but that refinement of its chemistry and further testing will be needed before it can...
A phase 2 trial of GSK2402968, designed to treat Duchenne MD caused by specific mutations, is now recruiting at 14 sites
Update (April 9, 2013): The U.S.-based phase 2 trial of drisapersen stopped recruiting new participants in January 2013. Results are expected in early 2014.
Update (Aug. 15, 2012): GlaxoSmithKline's exon-skipping drug GSK2402968 has been given a new generic name, "drisapersen."