~~<i id='1B3CD6942D'><strike id='1B3CD6942D'><tt id='1B3CD6942D'><pre id='1B3CD6942D'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

hotspot

leisure time

author：leisure time 2025-09-06 04:41:19 - browse：18222

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： Hepatitis C has a cure — but many Americans still lack access to it
Next article： Awakening the world to the whoosh: A patient's crusade

hotspot

focus

knowledge

entertainment

Wikipedia

SurveillancevideoofPhilanaHolmesreachingfortwoHappyMealsinthedrive-thruatMcDonald'sisshownonacourtro

WSS

Carbon Health diabetes program, a test of data

I learned firsthand about improving diversity in clinical trials

Hospitals struggle to profit from CAR

I learned firsthand about improving diversity in clinical trials

Tocagen's brain cancer gene therapy trial continues. Investors worry

Senate committee advances SCOTUS ethics bill after alleged justice misconduct

hotspot

leisure time

hotspot

DIEP, the 'gold standard' of breast reconstruction, is under threat

Divers poised to search for children washed away in Pennsylvania flash flood

Guidelines to prevent youth baseball injuries need more muscle

Stanford president faces new allegations over 1999 paper

Duchenne breakthrough therapy leaves behind pioneering families

Medicare’s new pay boost for primary care is angering specialists

How one medical school became remarkably diverse

NIH chief: government has fallen behind pharma on clinical trials

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

'Stay indoors': Escaped wild animal on the loose in Berlin prompts urgent citywide search

focus

DeSantis talks Trump, trans issues and 'what wokeness is'

EF3 tornado rips through North Carolina amid extreme weather nationwide

Readout Newsletter: Novo Nordisk trial, Verve base editing, etc

Healthy food access boosts outcomes in two studies

Driven by the pandemic, applicants flood public health schools

Experimental asthma app from Apple and Anthem shows promise

knowledge

entertainment

entertainment

Wikipedia