The Cooley’s Anemia Foundation (CAF) is offering support for significant travel to treatment centers for thalassemia patients. CAF offers limited financial assistance to individuals with thalassemia who experience financial hardship due to travel expenses incurred in obtaining an annual comprehensive care evaluation or extraordinary thalassemia care at a major treatment center. (Extraordinary thalassemia care shall […]
The FDA has approved Vertex and CRISPR’s gene-editing therapy Casgevy to treat beta thalassemia. Casgevy becomes the first therapy for the rare blood disorder to use the innovative CRISPR gene-editing technology. Vertex Pharmaceuticals and CRISPR Therapeutics have scored an FDA approval for their gene-editing therapy Casgevy (exa-cel) to treat transfusion-dependent beta thalassemia (TDT). The approval […]
Agios Announces Phase 3 ENERGIZE Study of Mitapivat Met Primary Endpoint and Both Key Secondary Endpoints in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia January 3, 2024 – Agios Pharmaceuticals, Inc. today announced that the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia achieved its primary endpoint of […]
