Opinion: Small study choices can decide Duchenne drug's fate
In a commentary, Mindy Leffler, whose son has Duchenne muscular dystrophy, argues that regulators can show flexibility in rare disease trials without compromising rigor. She emphasizes that countless minor decisions in study design can determine whether a treatment succeeds or fails.
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Original headline: “Opinion: Capricor’s Duchenne muscular dystrophy drug and the countless tiny decisions that can make or break a study.” Browse more stories.