The investigational compound is designed to raise levels of the SMN protein, a lack of which is the underlying cause of spinal muscular atrophy (SMA)
PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, says it will soon open a trial to test the safety and tolerability of its investigational drug RG7800 in adults and children with spinal muscular atrophy (SMA). The announcement was made in a Nov. 19, 2014, press release.
The European Medicines Agency expressed a 'positive opinion' on conditional approval for ataluren (Translarna) to treat Duchenne MD; a decision is expected within three months
The experimental Duchenne muscular dystrophy (DMD) drug ataluren (which has been given the brand name Translarna) has received encouragement from the European Medicines Agency (EMA) for conditional approval in European Union countries.
An increase in dystrophin was seen in a foot muscle in 61 percent of boys with nonsense-mutation DMD treated with ataluren in an MDA-supported study
An increase in levels of the dystrophin protein was seen after treatment with the experimental drug ataluren (originally called PTC124) in an MDA-supported, phase 2a, open-label trial of 38 boys with Duchenne muscular dystrophy (DMD) caused by a specific type of mutation in the dystrophin gene.
Although the experimental Duchenne-Becker MD drug ataluren appeared to benefit walking ability in a clinical trial, some researchers question whether it works via stop codon read-through
It's been widely accepted that the mechanism by which the experimental drug ataluren appears to benefit walking ability in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is that it causes "read-through" of premature stop codons — genetic instructions that cause cells to stop making a protein before the process is complete.
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...
The European Medicines Agency has agreed to review PTC Therapeutics’ application for conditional approval of ataluren, its experimental drug for DMD/BMD
Update (Dec. 6, 2012): This story has been updated with additional information about next steps for ataluren.