~~<i id='73B1A19789'><strike id='73B1A19789'><tt id='73B1A19789'><pre id='73B1A19789'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

focus

leisure time

author：Wikipedia 2025-09-27 19:21:30 - browse：3

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： Peter Hotez and the public health issue of online harassment
Next article： Roche's accidental data boosts hopes for anti

hotspot

Wikipedia

hotspot

focus

entertainment

SerialbiotechentrepreneurBobLangerwillserveasanadvisertoT.rxCapital.PatGreenhouse/GlobeStaffExecutiv

WSS

Wegovy improves function in certain heart failure patients

Abortion policy in the spotlight at Republican debate

Study: Spinal cord stimulation helps patients with paralysis walk again

Surge of respiratory disease in China has likely explanation

In key trial, Verve's treatment lowers cholesterol levels in patients

Readout Newsletter: Bayer study halts, Carmot to IPO, and more

focus

leisure time

hotspot

Hollywood unions extend contract negotiations for actors

Readout LOUD podcast: A CRISPR milestone, biotech mixology

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

Abortion policy in the spotlight at Republican debate

Walensky prepares to leave CDC — and Congress — behind

Weight loss drug costs may need more research, senator suggests

How to save PrEP access — and even expand it

FDA's Woodcock to retire early next year

Tori Bowie and the unacceptable rates of Black maternal death

California store owner shot dead in dispute over displaying Pride flag: Police

hotspot

Hospital at home shows how to bring innovation to health care

Why medical students are struggling with mental health

Abortion policy in the spotlight at Republican debate

Weight loss drug costs may need more research, senator suggests

NYU radiologists take on the challenge of expensive MRI machines

Morphosys mixed study results for blood cancer drug

Wikipedia

hotspot

focus

entertainment