~~<i id='BBBB42ED81'><strike id='BBBB42ED81'><tt id='BBBB42ED81'><pre id='BBBB42ED81'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

knowledge

leisure time

author：knowledge 2025-09-30 18:59:30 - browse：58

The Food and Drug Administration on Thursday approved a gene therapy to treat people with hemophilia A, an inherited and rare bleeding disorder.

The treatment, called Roctavian, is made by BioMarin Pharmaceutical. It was first approved in Europe in August 2022.

Roctavian is a one-time therapy that, in clinical trials, dramatically reduced bleeding episodes and helped patients live without the blood transfusions used to treat the disease. The FDA first rejected Roctavian in 2020, asking BioMarin to amass more data on its safety and long-term durability, and then delayed its decision again to account for additional evidence.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED Log In

Previous article： Wildfire smoke exposes gaps in outdoor worker protections
Next article： Researchers separate signal from noise in heart wearable data

hotspot

focus

knowledge

explore

leisure time

AdobeThekidneysaresomeofthemostarchitecturallycomplexorgansinthehumanbody—intricateinawaythatbecomes

WSS

States increasingly look to regulate hospital facility fees

Virginia high school admissions case could be legal follow

How one medical school became remarkably diverse

In age of alternative facts, a scholarly course on calling out crap

After affirmative action ruling, medical educators look to 'holistic review'

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

knowledge

leisure time

hotspot

Bright Health sells Medicare Advantage business to Molina

How dance can help health care workers heal from trauma

BioMarin wins approval for gene therapy to treat hemophilia A

Stanford updates investigation of president’s past research

BioMarin wins approval for gene therapy to treat hemophilia A

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

Drug repurposing or repositioning? The language matters

Gilgo Beach murders: What questions remain following Rex Heuermann's arrest?

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

New warning issued for rebooking air travel after delays, cancellations

focus

Ebola public health emergency is over, WHO declares

Moonlake's readout produced a cash windfall. Risks remain

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

At least 13 dead in Texas as scorching temps continue

States increasingly look to regulate hospital facility fees

Russia strikes Ukraine's critical port facilities in Odesa after halting grain deal

knowledge

knowledge

explore

leisure time