Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A.

Autor: Agarwal S; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Sandza K; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Obrochta Moss K; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Vora M; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Bowen A; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Bunch B; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Holcomb J; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Robinson TM; Late Clinical Development, BioMarin Pharmaceutical Inc, Novato, CA., Jayaram K; Pharmacovigilance, BioMarin Pharmaceutical Inc, Novato, CA., Russell CB; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Zoog S; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Vettermann C; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA., Henshaw J; Translational Sciences, BioMarin Pharmaceutical Inc, Novato, CA.

Publikováno v: Blood advances [Blood Adv] 2024 Sep 10; Vol. 8 (17), pp. 4606-4615.