focus -WSS

focus

author：focus 2025-09-27 13:04:01 - browse：388

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： Drug repurposing or repositioning? The language matters
Next article： Clinical trials giant ICON acquires HumanFirst

hotspot

WSS

FDA does about

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

Mifepristone protocols being wielded by pill's opponents in Supreme Court case

Abortion pill mifepristone to soon be dispensed at CVS, Walgreens

How to address the problem of discarded donor organs

Virtual repurposing can help discover new uses for existing drugs

knowledge

focus

entertainment

Supreme Court strikes down use of affirmative action

Children’s Hospital revives plan for pedestrian bridge

The high cost of giving birth even with insurance

Elisabeth Bik tackles the widespread issue of research misconduct

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

AstraZeneca loses challenge to Medicare drug price negotiation

Alkermes shareholders re

Kristina Burow of ARCH Venture Partners shares her next big bets STAT

George Santos makes 1st court appearance after pleading not guilty to 13 counts

Using force crossed my mind when examining a defiant young patient

focus

Study fuels hope CAR

5 ways to address the shortage of health care workers

Cholera vaccine supplies dangerously low, outbreaks surging

Change Healthcare cyberattack outage could last weeks

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

Change Healthcare cyberattack shows risk of industry consolidation

hotspot

focus

knowledge

knowledge