fashion -WSS

fashion

author：leisure time 2025-09-07 14:01:42 - browse：82264

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： Heart attack and stroke risk calculators need revising
Next article： Biotech stock slump in 2023: awaiting the turnaround

WSS

How Vertex's cystic fibrosis drug is changing patients' lives

Case for ALS therapy Nurown relies on ‘deficient’ data, FDA finds

Readout LOUD podcast: Live from the 2023 STAT Summit

Roivant's CEO is a star, but biotech's black hole persists

Emergency workers more likely to restrain Black patients: study

Ukraine and a new approach to mental health in war

hotspot

fashion

focus

Heart attack and stroke risk calculators need revising

CDC advisers recommend seasonal use of Pfizer RSV shot

Biden outlines 'new path' to provide student loan relief after Supreme Court rejection

How Vertex's cystic fibrosis drug is changing patients' lives

Alkermes shareholders re

STAT Summit: The economic case for universal basic health care

Moonlake's readout produced a cash windfall. Risks remain

STAT Summit: The economic case for universal basic health care

Anesthesiologist group: stop taking Ozempic before surgery

Roche drug slashes death risk in early

focus

How AI helped researchers track recovery from depression

Senators: Long

FDA panel to weigh approval of NurOwn, controversial ALS drug

It's been 20 years since Dolly the sheep. Where's my clone?

Intarcia’s device

STAT Virtual Event: Putting AI to the Test

focus

leisure time

entertainment

entertainment