science
Astellas to Restart Gene Therapy Trial for XLMTM Five Years After Patient Deaths Halted Program
Patients and families affected by the rare muscle disorder XLMTM are receiving a second chance at treatment as Astellas relaunches its gene therapy program. The move comes as fresh turmoil grips the top of the FDA with questions swirling over whether Commissioner Marty Makary will be ousted. STAT Plus also reported that Medicare is spending far less than expected on new Alzheimer’s drugs.
May 11, 10:52 AM·1m read1 source
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Patients and their families affected by XLMTM are getting a second chance at treatment as of May 11, 2026. Astellas is offering a resurrected gene therapy effort for the rare disease program on the same date. Five years after a gene therapy trial for the fatal muscle disorder XLMTM unraveled amid a string of devastating liver failure deaths, the community devastated by those losses now sees renewed hope.
The original trial's collapse left families with few options for the life-threatening condition that primarily affects young boys. Meghana Keshavan, a Biotech Correspondent who covers biotech and contributes to The Readout newsletter, reported the developments in an article published on May 11, 2026.
@statnews reported that the Astellas treatment offers new hope to the devastated rare disease community even as other challenges persist across biotech.
There is fresh turmoil at the very top of the FDA as of May 11, 2026. Questions swirled over whether FDA Commissioner Marty Makary will be ousted during the weekend prior to May 11, 2026. The same edition of The Readout carried a STAT Plus exclusive story on May 11, 2026 about Medicare spending far less than expected on new Alzheimer’s drugs.
That report highlighted underwhelming adoption of the therapies despite earlier expectations for broader uptake. The gene therapy revival arrives at a moment of both scientific and regulatory uncertainty. While the FDA faces leadership questions, the rare disease program that once ended in tragedy is now positioned for another attempt at delivering treatment to those who have waited years.
