focus -WSS

focus

author：explore 2025-09-30 13:14:09 - browse：45144

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： In Memoriam: Notable people who died in 2023
Next article： Physicians aren't 'burning out.' They're suffering from moral injury

knowledge

WSS

Lasker Awards 2023: Winners in biomedical research

STAT Virtual Event: Putting AI to the Test

Biotech stock slump in 2023: awaiting the turnaround

Microbiome research is starting to find links to chronic diseases

Senators: Long

FDA panel to weigh approval of NurOwn, controversial ALS drug

explore

focus

explore

Medicare details structure of new drug price negotiation program

It's been 20 years since Dolly the sheep. Where's my clone?

Virginia high school admissions case could be legal follow

Ukraine and a new approach to mental health in war

What does generative AI mean for health care? We asked experts

Lasker Awards 2023: Winners in biomedical research

New Eli Lilly Alzheimer’s data poses Medicare coverage conundrum

CDC recommends RSV monoclonal antibody for infants

In Memoriam: Notable people who died in 2023

How to ethically and practically extend health care to all

entertainment

Case for ALS therapy Nurown relies on ‘deficient’ data, FDA finds

Late stage cancers on the rise after pandemic delayed screenings

CDC recommends RSV monoclonal antibody for infants

Emergency workers more likely to restrain Black patients: study

Why are Kaiser Permanente staff considering a strike?

Pharma showers House GOP doctor with campaign cash

focus

knowledge

hotspot

entertainment