Biotech & Pharma

STAT+: Rare disease advocates fume over FDA's mixed signals

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The FDA rejected a gene therapy for Hunter Syndrome from Regenxbio, requiring more data that could delay approval by years; this decision is one of several recent rejections of rare disease treatments, raising concerns among advocates and executives about a shift towards stricter regulatory standards. For content creators, the key takeaway is the importance of highlighting the impact of regulatory decisions on patients with rare diseases and the potential for significant delays in accessing life-altering therapies.

Read the full article at STAT Pharma

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