Clinical trial shows some promise for Duchenne muscular dystrophy drug | DuchenneXchange

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Clinical trial shows some promise for Duchenne muscular dystrophy drug

UC Davis Neuromuscular Research Center

The results of an international clinical trial, led in part by a UC Davis School of Medicine expert, have shown that a drug therapy for a severe type of muscular dystrophy holds promise for a subgroup of patients.

In a paper just published online in the journal The Lancet, Craig McDonald and colleagues at 53 study sites in 18 countries describe the clinical benefit of using the drug ataluren for a certain group of patients carrying a specific “nonsense mutation” for Duchenne muscular dystrophy.

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