GENE THERAPY
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Patient-reported outcomes via MyCBDR and PROBE: essential to patient advocacy
Montreal, April 17, 2020 – In recent years, the creation of electronic tools that allow people with hemophilia (PwH) to report their health outcomes to...
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Gene Therapy Updates
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...
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Patients’ and caregivers’ “lived experience” crucial in CHS submissions on product access
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...