Homepage Bottom Left
-
OCTOSTIM SPRAY discontinued
Toronto, July 22, 2020 – Ferring Pharmaceuticals has discontinued production of OCTOSTIM SPRAY (150 mpg DIN 02237860) for the treatment of mild and moderate hemophilia A...
-
The WFH Gene Therapy and Bleeding Disorders Registries
WFH Gene Therapy Registry, presented by Dr. Barbara Konkle For many years gene therapy has been in the future – but now, for an increasing...
-
Two deaths in gene therapy trial for rare neuromuscular disease
The CHS recently learned of two deaths of children under five years old in a clinical trial using AAV8-based vectors in another rare disease area,...
-
Four-year follow-up of Biomarin’s FVIII gene therapy
Dr. John Pasi, Centre Director at the Royal London Haemophilia Centre, presented 4-year follow-up data on Biomarin Pharmaceutical’s Phase 1 /2 gene therapy trial of...
-
Redefining prophylaxis
A series of renowned speakers at the World Federation of Hemophilia Virtual Summit re-defined prophylaxis in this era of innovative therapies, including non-factor replacement therapies....