Sarepta Therapeutics demanded a prominent patient advocacy organization censor a video that contained pointed criticism of the company’s recently approved gene therapy for Duchenne muscular dystrophy, STAT has learned.
The incident raises questions about the financial ties between Sarepta and the advocacy group, Parent Project Muscular Dystrophy, and whether the drugmaker uses its money to influence, or even muzzle, debate over its Duchenne medicines. It has also bolstered concerns voiced by some parents of young adults with Duchenne that Sarepta is more focused on profiting from its gene therapy, called Elevidys, than providing them with information necessary to prove its efficacy and safety, despite the blessing of the Food and Drug Administration.
The video was recorded during a conference last month in Orlando sponsored by Parent Project Muscular Dystrophy, known colloquially as PPMD, and widely regarded as one of the largest and most influential Duchenne advocacy groups.
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