Tag: beta-thalassemia

THALASSEMIA CARE WALK 2024 – Locations & Info!

Thalassemia Care Walk

Care Walk is the Cooley’s Anemia Foundation’s annual fundraising event and opportunity to bring together the thalassemia community and its supporters around the country. Funds raised support medical research to fight thalassemia, a genetic blood disorder, as well as patient support services for thalassemia patients across the country. Click here to register for Care Walk!! […]

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FDA Approves Vertex & CRISPR’s Gene Editing Therapy to treat Beta Thalassemia

Treat Beta Thalassemia

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 […]

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Agios Pharmaceuticals Announces Phase 3 ENERGIZE – Treatment for Thalassemia Patients

Phase 3 ENERGIZE

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 […]

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Health Maintenance for Thalassemia Patients aged 50 Years and Older

Health Maintenance Award for Thalassemia Patients 50 years and older

The Cooley’s Anemia Foundation (CAF) recognizes the importance of maintaining health among thalassemia patients and is offering reimbursement of up to $500 for approved expenses related to health maintenance among U.S. thalassemia patients who are age 50 or older.

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FDA Approves bluebird bio’s ZYNTEGLO®, the First Gene Therapy for People with Transfusion-Dependent Beta-Thalassemia

zynteglo approved for beta-thalassemia

bluebird bio, Inc. announced the FDA has approved ZYNTEGLO®, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell transfusions.

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Roll of Beta-Thalassemia Gene Discovered

Researchers from Weill Cornell Medical College may have discovered the precise role of a gene in beta-thalassemia,  The study, entitled “Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in B-thalassemia”  was published in the May 14, 2008 online issue of the journal Blood, the official publication of the American Society of Hematology (ASH). Splenectomy (spleen removal) […]

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