Wikipedia -WSS

Wikipedia

author：fashion 2025-09-07 06:39:43 - browse：34158

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： BioMarin wins approval for gene therapy to treat hemophilia A
Next article： AstraZeneca chief proposes a novel edit to the drug pricing bill

focus

hotspot

WSS

RSV shot was 90% effective at preventing hospitalizations in kids

Headspace, Meta partner to launch VR meditation app for Quest

Readout Newsletter: Acadia, Eli Lilly, Madrigal Pharma updates

Stem cell treatment guidelines are released by FDA

Amylyx ALS drug fails large clinical trial

Snackable artificial intelligence, expert AI, and the pharma industry

focus

Wikipedia

focus

Medical leaders decry Supreme Court decision on affirmative action

What American doctors can learn from their Israeli counterparts

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

In his State of the Union, Biden takes clear aim at 'Big Pharma'

United colonoscopy coverage change 'may cost lives,' doctors say

A deep dive into Applied Therapeutics’ data, and questions on Cassava

The cancer drug shortage isn’t new — and neither are the solutions

Novo Nordisk obesity pill bests Wegovy in early trial

Guidelines to prevent youth baseball injuries need more muscle

Headspace, Meta partner to launch VR meditation app for Quest

Wikipedia

IVF, abortion rights issues kick off Biden’s State of the Union

Advances in CAR

Readout Newsletter: Amylyx, Alto Neuro, and Eli Lilly for the Oscars

'Make People Better': The CRISPR babies story you probably don't know

In surprise move, FDA calls for advisory committee on Lilly's Alzheimer's drug

First medicine developed to treat MASH expected to be approved soon

focus

hotspot

explore

hotspot