Gene Therapy for Hemophilia Becomes a Reality
Uptake of new options for hemophilia A and B has been a bit slow
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Uptake of new options for hemophilia A and B has been a bit slow
Patients who received lovo-cel had durable remissions and reductions in vaso-occlusive events, pain crises, and hospitalizations in the longest running gene therapy trial for sickle cell disease.
More than half of children in study had no treated bleeds at follow-up of more than 100 weeks
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Dec 9, 2023--
– Phase III HAVEN 7 primary data presented at ASH 2023 provide additional confidence in the favorable efficacy and safety profile of subcutaneous Hemlibra given soon after birth – – At nearly...