entertainment -WSS

entertainment

author：leisure time 2025-10-02 05:38:55 - browse：2

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： United colonoscopy coverage change 'may cost lives,' doctors say
Next article： What would happen to your eyes if you looked at the solar eclipse?

focus

WSS

Why are elite athletes different? Take a look at their microbes

What is the Wagner Group? The 'brutal' Russian military unit in Ukraine

3 hurdles to bringing medical devices to the U.S. market

3 hurdles to bringing medical devices to the U.S. market

He's 19 months old and has one of the world’s rarest diseases

Longer MASH drug regimen improved liver, Akero study finds

focus

entertainment

entertainment

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

Medicare drug price negotiation hearing pits pharma v Biden admin

Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

Gene therapy has helped boys walk. Can scientists keep it that way?

Cancer drug shortages should be causing more outrage

Readout Newsletter: BridgeBio, Akero, and human embryo models

Anesthesiologist group: stop taking Ozempic before surgery

The public health thinking behind the CDC’s new Covid guidance

DIEP, the 'gold standard' of breast reconstruction, is under threat

Longer MASH drug regimen improved liver, Akero study finds

focus

Lack of health data on LGBTQ+ people has serious consequences

Weight bias in eating disorder treatment complicated by new weight loss drugs

Cloning humans is technically possible. It's curious no one has tried

Cloning humans is technically possible. It's curious no one has tried

First over

New guidelines on severe brain injury complicate already difficult decisions

knowledge

Wikipedia

focus

knowledge