WebMD

Altuviiio for Hemophilia A

People with severe hemophilia A are at risk for prolonged bleeding events that can cause serious complications. To prevent these bleeding events, most people with this condition get injections of a ...
The New England Journal of Medicine

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Valoctocogene roxaparvovec delivers a B-domain–deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A. The findings of a ...
Medscape

Gene Therapy for Hemophilia A 'Transforming and Liberating'

A form of gene therapy has shown significant benefit in men with a severe form of the bleeding disorder hemophilia A. "If approved, this first-generation gene therapy would offer a new choice for care ...
Healio

Gene therapy increases factor VIII activity, reduces bleeding in severe hemophilia A

Please provide your email address to receive an email when new articles are posted on . Valoctocogene roxaparvovec induced endogenous factor VIII production and significantly reduced bleeding and ...

Michigan man gets new Roctavian gene therapy for hemophilia A

Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene therapy for severe hemophilia A.
Medscape

Hemophilia A Gene Therapy Durable at 2 Years; Under FDA Review

Clinical data have been published for a gene therapy for hemophilia A that is approaching the market — valoctocogene roxaparvovec (Roctavian), which is currently under review by the US Food and Drug ...
Healio

Investigational gene therapy leads to sustained factor VIII expression in hemophilia A

Please provide your email address to receive an email when new articles are posted on . The novel gene therapy SPK-8011 induced sustained factor VIII expression in patients with hemophilia A, ...
MedPage Today

Gene Tx Shows Sustained Bleeding Control in Severe Hemophilia A at 2 Years

A single infusion of the gene therapy valoctocogene roxaparvovec led to significantly reduced bleeding rates that persisted in men with severe hemophilia A, 2-year results from the phase III GENEr8-1 ...
MedPage Today

Year in Review: Hemophilia

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window Over the past ...
STAT

Inspired by the determination of people with hemophilia A living with the burden of severe bleeds

Christine Orr, a self-described “hemophilia mom,” is the primary caregiver for her ten-year-old son John. “I’m the only one who treats him,” she says. Upon learning that John had severe hemophilia A, ...
Nasdaq

BioMarin Announces New England Journal of Medicine Publishes 3 Years of Follow-up Data in Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

SAN RAFAEL, Calif., Jan. 2, 2020 /PRNewswire/ --BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the New England Journal of Medicine (NEJM) published an independently peer-reviewed ...