~~<i id='A50F634437'><strike id='A50F634437'><tt id='A50F634437'><pre id='A50F634437'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

entertainment

explore

author：explore 2025-10-02 00:41:00 - browse：53

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： Virginia high school admissions case could be legal follow
Next article： UPS workers' union leader explains what's at stake as strike edges closer

knowledge

hotspot

entertainment

focus

5:55TheTitansubmersible,operatedbyOceanGateExpeditionstoexplorethewreckageofthesunkenSSTitanicoffthe

WSS

Sen. Bill Cassidy on how to lower drug prices

Zelenskyy on how Russian invasion may end, gratitude for US aid and Putin's weakness

Tennessee woman gets emergency hysterectomy after doctors deny early abortion care

In medicine, common shorthands can shortchange care

Neutralizing antibodies against HIV shows promise in children

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

entertainment

explore

knowledge

State Dept. review finds Biden bears some blame for Afghanistan failures

Moderna hits safety problems in bold bid to reinvent medicine

New Eli Lilly Alzheimer’s data poses Medicare coverage conundrum

North Korean leader's powerful sister says warplanes repelled US spy plane

Drug repurposing or repositioning? The language matters

Drinking water from half of US faucets contains PFAS: Study

Affirmative action in medical school literally saved lives

Kansas must stop changing trans people's sex listing on driver's licenses, judge says

New Eli Lilly Alzheimer’s data poses Medicare coverage conundrum

Democratic lawmaker calls for 'well

hotspot

The PBM ad wars are taking over Washington, D.C.

Moderna hits safety problems in bold bid to reinvent medicine

Trump lawyer welcomes Pence testifying in Jan. 6 trial, won't say if Trump will too

The UK government moves asylum

How corporate America is slashing DEI workers amid backlash to diversity programs

South Korea begins evacuating thousands of Scouts from coastal campsite as tropical storm nears

hotspot

entertainment

entertainment

focus