Some boys had astonishing recoveries. But other boys died. Now Astellas is returning to revised gene therapy for XLMTM.
By Jason Mast
May 11, 2026
Jason Mast
jason.mast@statnews.com
Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77.
Doctors have linked a boy's tumor to a gene therapy, but say the rare risks need to be weighed against the profound benefits such…
Alicia and Jon Langenhop's three children were each diagnosed with a rare disorder. A clinical trial was "a no-brainer."
“We’re still very much in the early stages,” a gene therapy expert said. “But every day, he’s showing us signs that he’s growing…
Medicine is undergoing a profound transformation by shifting focus from populations to individual patients. A novel custom drug…
Alzheimer's drug rollout letdown, a China-based heart drug startup, and more biotech news from The Readout
Blood Cancer United Acquires Investigational Drug Supply to Preserve Access for Children with Rare, Fatal Leukemia
