PTC, Genzyme muscular dystrophy drug falls short
PTC Therapeutics Inc. and Genzyme Corp. said Wednesday their potential muscular dystrophy treatment did not meet its primary goal in a midstage study.
In the study, ataluren failed to meet its goal of improving walking distance. Muscular dystrophy is a genetic condition that weakens the muscles and hampers walking ability. Results did show that the drug was well tolerated, the companies said.
The results of the 48-week study are preliminary, the companies said.
Ataluren is also being studied as a potential treatment for a form of cystic fibrosis and hemophilia.
Shares of Genzyme, based in Cambridge, Mass., rose 36 cents to $57.18 in morning trading. PTC is based in South Plainfield, N.J.