comprehensive -WSS

comprehensive

author：hotspot 2025-09-02 11:45:37 - browse：542

The FDA building -- First Opinion coverage from STAT — Adobe

The Food and Drug Administration on Monday approved a gene therapy for metachromatic leukodystrophy, a devastating genetic disorder that eats away at affected children’s neurons.

The medicine, marketed as Lenmeldy, is the first treatment for MLD, which is otherwise fatal. Lenmeldy has to be given before symptoms appear or, in some cases, after the first signs of disease, so it won’t be helpful for many patients living with MLD today. But when given in time, it appears to be near-curative, allowing some children to grow up and attend school with unaffected peers.

Lenmeldy is already marketed in Europe, under the name Libmeldy, and has helped a handful of patients in the U.S. who were treated under compassionate use, as their doctors feared they didn’t have time to wait for approval.

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

Subscribe Log In

Previous article： Medicare details structure of new drug price negotiation program
Next article： What is artificial intelligence? Experts weigh in

WSS

What is the Turing Test that determines if computers can think?

New chief aims to remake Sanofi Genzyme

Florida babysitter arrested over hot car death, authorities say

Bursting ice dam in Alaska highlights risks of glacial flooding around the globe

3 dead in firefighting helicopter crash in California after midair collision with 2nd helicopter

Pfizer's RSV vaccine recommended for approval in Europe

Wikipedia

comprehensive

leisure time

How to save PrEP access — and even expand it

Listen: BridgeBio's big week & Lilly's Alzheimer's data

The high cost of giving birth even with insurance

What is the Turing Test that determines if computers can think?

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

Why the advice to take all your antibiotics may be wrong

Medical records are filled with copy

Moderna hits safety problems in bold bid to reinvent medicine

23andMe had bad news about my health. I wish a person had delivered it

What is the Turing Test that determines if computers can think?

focus

Florida babysitter arrested over hot car death, authorities say

New chief aims to remake Sanofi Genzyme

New chief aims to remake Sanofi Genzyme

Judges halt a Biden rule offering student debt relief for those alleging colleges misled them

Study: Misdiagnosis causes 800,000 deaths, serious disabilities a year in U.S.

Thousands in Haiti march to demand safety from violent gangs as killings and kidnappings soar

entertainment

focus

focus

entertainment