We’re proud to announce five new Cooley’s Anemia Foundation Medical Research Fellowships for the 2024-2025 grant cycle. The total amount of funding for the research Fellowships is $250,000. These Fellowship recipients were assessed on the basis of the quality of the scientific content, the academic accomplishments and future promise of the investigator, the quality of […]
*IMPORTANT: The following information is a press release that has been released in advance of the date being presented for peer review* Agios Pharmaceuticals announced Monday that its drug Pyrukynd (mitapivat), an oral pyruvate kinase activator, reached its primary endpoint in a Phase III trial evaluating adults with the blood disorder transfusion-dependent alpha- or beta-thalassemia. […]
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!! […]
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 […]
The council will bring together patients, caregivers and physicians from these communities, which share commonalities in underlying disease pathology, difficulties in transitioning from pediatric to adult care and quality of life concerns.
Are you or a loved one considering gene therapy as a treatment option for transfusion dependent beta thalassemia? Watch the recording of the Gene Therapy Webinar featuring Dr. Sujit Sheth and reps from bluebird bio now!
The Eleventh Cooley’s Anemia Symposium will bring together basic scientists, clinical investigators, and clinicians to review and discuss recent research for thalassemia syndromes.
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.
March 31, 2021 – The Cooley’s Anemia Foundation and Acceleron Pharma Inc. are offering up to four college scholarships of $5,000 each to individuals with a clinically significant form of thalassemia (alpha or beta thalassemia major, beta thalassemia intermedia, e beta thalassemia, hemoglobin H disease or hemoglobin H disease Constant Spring). Two of these scholarships […]
