Hope, Evidence, and Hard Questions in Duchenne Muscular Dystrophy

I’ve been reflecting on the disappointing outcomes and the reaction to Sarepta Therapeutics’ recently announced clinical trial results for its Duchenne muscular dystrophy treatments, AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen). The tension is evident: Families want treatments that benefit patients, while critics note the evidence reveals a different story. Duchenne muscular dystrophy is a relentless disease. When a company suggests it […]

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