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Rare disease advocates fume over FDA’s mixed signals

FDA has rejected or reversed course on five gene therapies for rare diseases while simultaneously unveiling expedited pathways for new treatment approvals.

BusinessBy Wire ServicesFebruary 26, 20261 min read

Last updated: April 3, 2026, 8:27 PM

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Rare disease advocates fume over FDA’s mixed signals

FDA has rejected or reversed course on five gene therapies for rare diseases while simultaneously unveiling expedited pathways for new treatment approvals.

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Wire Services

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