British drug maker
The companies said the potential treatment, drisapersen, did not meet the study's main goal of generating a statistically significant improvement in a six-minute walking distance test compared to the placebo. Researchers also saw no difference in some secondary assessments of motor function like a walk/run test and a stair climb.
The study involved a total of 186 boys, and the company expects to evaluate the results some more before determining a next step for the potential treatment.
Duchenne muscular dystrophy is a rare and fatal genetic disease that causes increasing muscle weakness and affects one of every 3,500 boys born worldwide.
Drisapersen is not approved for use anywhere in the world.
U.S.-traded shares of
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