GENE THERAPY

Patient-reported outcomes via MyCBDR and PROBE: essential to patient advocacy
David Page on 07/04/2020
Montreal, April 17, 2020 – In recent years, the creation of electronic tools that allow people with hemophilia (PwH) to report their health outcomes to...
Gene Therapy Updates
David Page on 07/04/2020
FDA grants priority review for Biomarin’s hemophilia A gene therapy San Rafael, California, February 21, 2020 – Biomarin Pharmaceuticals has announced that the U.S. Food...
Patients’ and caregivers’ “lived experience” crucial in CHS submissions on product access
David Page on 07/04/2020
It’s an exciting time in care for people with bleeding disorders. In recent years, a number of novel therapies have come through clinical trials and...

Patient-reported outcomes via MyCBDR and PROBE: essential to patient advocacy