Wikipedia -WSS

Wikipedia

author：explore 2025-09-27 12:22:10 - browse：4

A gene therapy study, halted when four children with a rare neuromuscular disorder died after treatment, showed enough promise to merit finding a path forward, according to the medicine’s manufacturer.

The treatment, made by the Japanese pharmaceutical company Astellas, led to severe and fatal liver problems for four of the 24 treated children with X-linked myotubular myopathy, or XLMTM, a genetic disease that severely degrades muscle function and kills most patients before the age of 10. Three died in 2020; the fourth died a year later.

In an analysis of its clinical trial, published in Lancet Neurology on Wednesday, Astellas observed dramatic benefits in the study’s surviving patients and a potential explanation for the deaths that derailed the trial. The hope, for Astellas and for families affected by XLMTM, is that the company can find a safe way to proceed and convince the Food and Drug Administration to allow it to resume development.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Medicare proposal to cover medical devices draws a mixed reaction
Next article： Readout Newsletter: Moderna bird flu vaccine, Biohaven, ASCO

WSS

Immunocompromised patients offer clues on chronic Covid

Sanofi to acquire U.S. biotech Inhibrx in $2.2 billion deal

Childbirth

What causes osteoarthritis? Researchers study link to brain protein

To bolster infectious disease surveillance, genomics powerhouses team up

Latest bird flu infection made farmworker sicker than earlier cases

focus

Wikipedia

focus

George Santos makes 1st court appearance after pleading not guilty to 13 counts

Prior authorization reform is both possible and imperative

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

Lab trade group sues FDA to halt regulation of lab

Hepatitis C has a cure — but many Americans still lack access to it

High breast density mammograms need follow

After affirmative action ruling, medical educators look to 'holistic review'

What will save rural psychiatry services? Higher Medicaid payments

Malaria cases in Florida and Texas: Here’s what you need to know

Inaction leaves children at risk from dangerous chemotherapy drug

explore

Adam's Biotech Scorecard: Do Sarepta's exon

AI used to predict Covid

Health insurers say they are turning to AI to cut costs, improve care

Report: NIH

Adam's Biotech Scorecard: Do Sarepta's exon

Readout Newsletter: Parkinson's study, Gilead, CG Oncology

hotspot

entertainment

entertainment

focus