Sarepta’s Muscular Dystrophy Drug Improves With Time; Stock Booms
Summary
That was a statistically significant improvement compared with boys on the placebo, who lost 78 meters of walking ability over the same period of time, Sarepta said.The 6-minute walk test isn’t a perfect measurement of clinical success, although the FDA has used it many times for trials of drugs for rare diseases.Back then, the company reported its drug was able to help boys produce 22.5 percent of the normal levels of the dystrophin protein they need for muscle function—a scientifically tantalizing result.Based on what it has learned so far from the muscle biopsy tests, which show that it can take 12 to 24 weeks for patients to start building up their dystrophin levels, Sarepta is betting that there’s a delayed effect working in its favor.Any whiff of progress Sarepta makes is bound to attract significant attention from patient advocates, who are crying out for some good news for the treatment of this disease.