focus -WSS

focus

author：explore 2025-09-30 16:26:16 - browse：57

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： State Dept. review finds Biden bears some blame for Afghanistan failures
Next article： How doctors’ personal politics affect patients

focus

WSS

'Investigate these claims': UFO transparency at center of House hearing

Medical school prerequisites are outdated

Readout Newsletter: Novo Nordisk trial, Verve base editing, etc

Bronny James, LeBron's son, suffers cardiac arrest during basketball practice at USC

Device maker Teleflex to acquire urology company for $600M

White House drug czar issues call for 'recovery

entertainment

focus

explore

Over 800 arrested across France in 3rd night of riots after fatal police shooting of teen

STAT Report: Patient voices and perspectives

How one medical school became remarkably diverse

Senate panel puts off hardest PBM reforms for another day

Alkermes shareholders re

'Investigate these claims': UFO transparency at center of House hearing

In Memoriam: Notable people who died in 2023

Bronny James, LeBron's son, suffers cardiac arrest during basketball practice at USC

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

CDC Director Mandy Cohen on transparency, trust, and health

focus

FDA cancer chief on how to get things done: ‘Don’t tell anybody'

Roche’s Alnylam deal shows drug firms interested in heart drugs

Readout Newsletter: Zepbound, Valneva's vaccine, and more

Ventyx halts psoriasis drug development after lackluster trial results

Rise in cancer among younger people worries and puzzles doctors

Readout Newsletter: Ozempic panic wanes, Legend x Novartis, etc.

hotspot

focus

leisure time

hotspot