~~<i id='7B7FE7C929'><strike id='7B7FE7C929'><tt id='7B7FE7C929'><pre id='7B7FE7C929'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

leisure time

hotspot

author：focus 2025-09-27 10:00:48 - 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： Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies
Next article： Organ transplantation is at a crossroads. Major reform is needed

focus

leisure time

hotspot

entertainment

knowledge

leisure time

AstudyfromZiyadAl-Aly,clinicalepidemiologistatWashingtonUniversityinSt.Louis,showedthatvaccinationap

WSS

What Congress can do to address the rural doctor shortage

Russian special forces kill a gunman who broke into a private house near Moscow

Drug for rare kidney disease narrowly misses mark in clinical trial

HIPAA doesn't protect reproductive records from prosecutors

CDC backtracks, saying fully vaccinated people can go maskless indoors

Why are Kaiser Permanente staff considering a strike?

leisure time

hotspot

focus

The cancer drug shortage isn’t new — and neither are the solutions

Southern California school board OKs curriculum after Gov. Gavin Newsom threatened a $1.5M fine

New Eli Lilly Alzheimer’s data poses Medicare coverage conundrum

Organ transplantation is at a crossroads. Major reform is needed

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

Why are Kaiser Permanente staff considering a strike?

Psychedelics group wrestles with new pharma identity

Providers still hesitate to prescribe buprenorphine, study finds

Medicare proposal to cover medical devices draws a mixed reaction

Microbiome research is starting to find links to chronic diseases

leisure time

Organ transplantation is at a crossroads. Major reform is needed

Drug for rare kidney disease narrowly misses mark in clinical trial

Hyaluronic acid injections don’t help knee osteoarthritis, study finds

7 of the most gruesome medical devices in history

Death toll from an explosion and collapse of a Paris building in June climbs to 3

Senate HELP committee spars over health care workforce policies

hotspot

entertainment

knowledge

leisure time