entertainment -WSS

entertainment

author：hotspot 2025-10-03 00:39:56 - 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： DIEP, the 'gold standard' of breast reconstruction, is under threat
Next article： Chris Christie meets with Zelenskyy during Ukraine trip

focus

WSS

NVIDIA and Google execs on the future of AI and drug discovery

Can we make enough H5N1 bird flu vaccine if there's a pandemic?

Flint parents haunted by uncertainty, fears of lead poisoning

With measles on the rise, a personal story of a devastating disease

2 officers injured in shooting in Orlando, police say

Bird flu: Don't repeat Covid's 'health versus the economy' framing

focus

entertainment

hotspot

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

Biotech startup Celsius cuts 75% of staff ahead of first trial start

How to save PrEP access — and even expand it

Cytokinetics CEO stresses company can launch heart drug on its own

The high cost of giving birth even with insurance

Analysis of pharma R&D ranks Novo Nordisk, J&J above their peers

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

Cytokinetics CEO stresses company can launch heart drug on its own

Alkermes shareholders re

Addiction experts warn against a second 'war on drugs'

entertainment

Venture capitalist Bob Kocher on the state of digital health

Late stage cancers on the rise after pandemic delayed screenings

Readout LOUD podcast: Live from STAT Breakthrough Summit West

Addiction experts warn against a second 'war on drugs'

When prostate cancer spreads, where it goes matters

Judge partially blocks Texas abortion ban for medical emergencies, fatal diagnoses

Wikipedia

focus

leisure time

leisure time