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Pfizer Prospective Treatment for Duchenne Muscular Dystrophy Fails to Meet Goals
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Pfizer Prospective Treatment for Duchenne Muscular Dystrophy Fails to Meet Goals
Jun 12, 2024 3:02 PM

05:30 PM EDT, 06/12/2024 (MT Newswires) -- Pfizer ( PFE ) late Wednesday said a late-stage trial of its fordadistrogene movaparvovec candidate in young boys with Duchenne Muscular Dystrophy did not meet the primary and secondary endpoints.

The trial sought to compare fordadistrogene movaparvovec with placebo in boys aged four to seven years with Duchenne, one year after the treatment for the primary goal of improved movement, as measured by North Star Ambulatory Assessment.

The company did not immediately disclose specific results, but said key secondary endpoints also did not demonstrate a significant difference from placebo.

The safety profile of the product candidate was manageable, with mostly mild to moderate adverse events. Treatment-related serious adverse events generally responded to clinical management.

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