Dr. Marion Dugdale Championed Hemophilia Patients in Clinics and Classrooms

Dr. Marion Dugdale championed hemophilia patients in clinics and classrooms

She set up her lab in an empty room. With cobbled equipment and the blood of cows, dogs and humans, she set out to work on advances in hemophilia.

Roctavian Gene Therapy Controls Bleeding for Five Years in Severe Hemophilia A

BioMarin's Roctavian gene therapy shows long-term effectiveness in treating severe hemophilia A, reducing bleeding and improving quality of life for patients.

Roctavian sustains bleed control, maintains factor VIII for 5 years

A single dose of Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for severe hemophilia A from BioMarin Pharmaceutical, maintains factor VIII (FVIII) levels at near-normal or normal levels for up to five years, with most patients staying off prophylaxis, or preventive treatment. These lasting benefits were observed in GENEr8-1 (NCT03370913), a global Phase 3 clinical study. The study showed Roctavian reduced the need for regular FVIII…