nonambulatory

A study of exon-skipping drug GSK2402968 in nonambulatory subjects with DMD has opened at Nationwide Children's Hospital in Ohio

posted on June 18, 2010 - 11:58am
Update (Nov. 5, 2012): Results announced in November 2012 showed the drug, now called drisapersen, reached blood levels approximately proportional to the injected dose at two of the three dosage levels tested and was not associated with serious adverse events. See Drisapersen Appears Safe in Non-Walking Boys With DMD.

The experimental drug ataluren, developed to overcome nonsense mutations in Duchenne and Becker MD, did not meet its primary end point in a large-scale human trial

posted on March 3, 2010 - 4:55pm
The biopharmaceutical firm PTC Therapeutics announced March 3 that ataluren, its experimental drug for certain forms of Duchenne (DMD) and Becker (BMD) muscular dystrophy, although safe and well tolerated, failed to meet its primary end point within the 48-week duration of the phase 2b trial. That end point was an improvement in how far boys with DMD or BMD could walk in six minutes.

PTC Therapeutics, with support from MDA, is testing ataluren in boys with DMD or BMD who have nonsense mutations and are no longer walking.

posted on January 19, 2010 - 9:33am
Update (March 3, 2010): An update to the article Ataluren Results Disappointing was posted.