fashion -WSS

fashion

author：entertainment 2025-09-29 21:26:13 - browse：566

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： Apple is now the first public company to be valued at $3 trillion
Next article： Walensky prepares to leave CDC — and Congress — behind

hotspot

entertainment

WSS

WATCH: Why do our ears pop?

Weight loss drugs aren’t covered by Obamacare. That may change

Congress may force more Covid

Congress may force more Covid

The high cost of giving birth even with insurance

Medicare details structure of new drug price negotiation program

focus

fashion

leisure time

Sports medicine is finally prioritizing gender equality

Dobbs anniversary: the lost opportunity of abortion as health care

Duchenne breakthrough therapy leaves behind pioneering families

WATCH: Why do our ears pop?

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

Flagship Pioneering startup Sonata Therapeutics lays off 21

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

Flagship Pioneering startup Sonata Therapeutics lays off 21

Peter Hotez and the public health issue of online harassment

What does generative AI mean for health care? We asked experts

entertainment

Researchers tackle phage therapy hurdles for wider adoption

Dobbs anniversary: the lost opportunity of abortion as health care

Readout Newsletter: Sarepta, Alnylam, and Royalty Pharma

New CMS data security rules will hurt Medicare, Medicaid research

Researchers tackle phage therapy hurdles for wider adoption

The high cost of giving birth even with insurance

Wikipedia

hotspot

entertainment

entertainment