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

STAT PlusNewsletterThe Readout Astellas treatment offers new hope to a devastated rare disease community Alzheimer’s drug rollout letdown, a China-based heart drug startup, and more biotech news from The Readout Manage alerts for this article Email this article Share this article By Meghana KeshavanMay 11, 2026 Biotech Correspondent Meghana Keshavan[email protected]Meghana Keshavan covers biotech and contributes to The Readout newsletter. Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

There’s fresh turmoil at the very top of the FDA: All weekend, questions have swirled over whether Commissioner Marty Makary will be ousted.Advertisement Also, we have a resurrected gene therapy effort in a once-devastated rare disease program, and underwhelming Alzheimer’s drug adoption. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts.

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2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, drug development, Drug prices, research Submit a correction requestReprints Meghana Keshavan Biotech Correspondent Meghana Keshavan covers biotech and contributes to The Readout newsletter. Newsletter Understand how science, health policy, and medicine shape the world every day Recommended Health May 11, 2026 STAT Plus: Five years after disaster, a rare disease community gets new chance at treatment Exclusive May 11, 2026 STAT Plus: Medicare is spending far less than expected on new Alzheimer’s drugs Advertisement Biotech May 8, 2026 The biotechs wear Prada The Readout May 8, 2026 STAT Plus: The biotech news you need to read today The Readout May 7, 2026 STAT Plus: FDA revisits a rare cancer treatment it rejected a few months ago Subscriber Picks