leisure time -WSS

leisure time

author：Wikipedia 2025-09-30 00:46:34 - browse：1

Cambridge: Biogen — Ruby Wallau for STAT

A plan to use new genetic techniques to treat non-genetic forms of ALS failed in an early study, Ionis and Biogen announced Thursday. The companies said they would discontinue the program.

In the randomized, 99-person trial, the companies tested a drug designed to silence a gene called ATXN2. Over the last decade, numerous drugs have been built to silence genes in ALS patients, but most of them have, fittingly, focused on rare genetic forms of ALS, where neurons die largely because of the work of a single mutated stretch of DNA.

This approach has led to an approved drug for one very small subset of ALS cases, driven by the gene SOD1, and drugs for other genes are in trials. But only perhaps 10% — or at most 20% — of ALS cases are genetic. The hope, based on some genetic data and promising mice experiments, was that lowering ATXN2 would help any patient who develops ALS.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Monthly

$39

Totals $468 per year

$39/month Get Started

Totals $468 per year

Starter

$30

for 3 months, then $39/month

$30 for 3 months Get Started

Then $39/month

Annual

$399

Save 15%

$399/year Get Started

Save 15%

11+ Users

Custom

Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users

$300

Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： What does generative AI mean for health care? We asked experts
Next article： Senate committee scrutinizes Medicare Advantage brokers

hotspot

WSS

UnitedHealth’s board chair also owns a personal investment firm

CEOs share strategies for surviving biotech slump

RSV vaccines may be linked to small increased risk of developing GBS

Obesity now affects over 1 billion people worldwide, study finds

ALS groups 'won't be played again' by the FDA, drug sponsors

International team creates first chimeric human

focus

leisure time

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

entertainment

Trump demands the U.S. pay no more for drugs than other countries … again

FDA warns J&J's Abiomed: We will regulate your software

Guidelines to prevent youth baseball injuries need more muscle

How to fix the rural physician shortage in the U.S.

State Dept. review finds Biden bears some blame for Afghanistan failures

Covid isolation guidance eased by the CDC

Continuity nursing helped my family in our darkest PICU moments

Researchers explore possible ties between long Covid, menopause

Medicare details structure of new drug price negotiation program

Readout Newsletter: Ironwood, Pfizer, FogPharma updates

knowledge

Senate committee scrutinizes Medicare Advantage brokers

How climate

RaDonda Vaught to speak at CommonSpirit event on patient safety

FDA warns J&J's Abiomed: We will regulate your software

FogPharma raises $145 million in rare Series E round

Why health workers in Gaza are injured or die at high rates

explore

hotspot

hotspot

focus