Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.

Margareth C OzeloJohnny MahlanguK John PasiAdam GiermaszAndrew D LeavittMichael LaffanEmily SymingtonDoris V QuonJiaan-Der WangKathelijne PeerlinckSteven W PipeBella MadanNigel S KeyGlenn F PierceBrian O'MahonyRadoslaw KaczmarekJoshua HenshawAdebayo LawalKala JayaramMei HuangXinqun YangWing Y WongBenjamin Kimnull null

Published in: The New England journal of medicine (2022)

In patients with severe hemophilia A, valoctocogene roxaparvovec treatment provided endogenous factor VIII production and significantly reduced bleeding and factor VIII concentrate use relative to factor VIII prophylaxis. (Funded by BioMarin Pharmaceutical; GENEr8-1 ClinicalTrials.gov number, NCT03370913.).

Keyphrases

early onset
genome wide
copy number
genome wide identification
smoking cessation