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
posted on July 3, 2013 - 3:37pm
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.
The 62-week results from a phase 2b trial of eteplirsen show the drug continued to stabilize walking ability in boys with Duchenne MD
posted on December 7, 2012 - 4:57pm
Eteplirsen, an exon-skipping drug in development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show benefit at 62 weeks with respect to the distance walked in six minutes.