An experimental drug appears to preserve and possibly even improve the ability of boys with Duchenne muscular dystrophy to walk, according to the results of a clinical trial announced on Wednesday, raising hopes that the first effective treatment for the disease may be on the horizon.
Boys with the disease who received the highest dose of the drug had a slightly improved ability to walk after 48 weeks of treatment, the drug’s developer, Sarepta Therapeutics, announced. By contrast, the boys who received a placebo suffered a sharp decline in how well they could walk.
The drug, called eteplirsen, also appeared to restore levels of the key protein that muscular dystrophy patients lack to about half of normal levels, Sarepta said.
Read it all. The blog has been following this story for a while now–keep your eye on it, it is a potnetially once in a lifetime event; KSH.