STAT+: Astellas treatment offers new hope to a devastated rare disease community

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There’s fresh turmoil at the very top of the FDA: All weekend, questions have swirled over whether Commissioner Marty Makary will be ousted.

Also, we have a resurrected gene therapy effort in a once-devastated rare disease program, and underwhelming Alzheimer’s drug adoption. 

Continue to STAT+ to read the full story…