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Biomarin’s gene therapy for hemophilia A to be called Roctavian
Montreal, May 14, 2020 – Biomarin announced on May 11 its gene therapy currently awaiting approval by the U.S. Food and Drug Administration will be...
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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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Updated Standards of Care sent for endorsement
Montreal, April 17, 2020 – The Canadian Integrated and Comprehensive Care Standards for Bleeding Disorders have now been finalized and sent to stakeholders for endorsement....
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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...