This blog will be a place for the Cooley’s Anemia Foundation to share information from and thoughts of invited members of the thalassemia community.
*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 […]
We are delighted to share with you TIF’s latest publication for patients living with thalassemia, sickle cell disease (SCD) and other rare anemias and their families entitled ‘Travel Guidance for Patients and Families’.
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.
The Cooley’s Anemia Foundation’s Westchester-Rockland Chapter welcomes all to experience its 25th annual “Night at the Races” benefit on April 1, 2023.
When Maybelline, the iconic beauty brand behind Maria’s first blush, invited her to star in their “We Speak” disability pride campaign, she seized the opportunity to become the very thing she longed to see as a child—a model proudly representing the thalassemia community.
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 2, 2017- CAF launched our “Volunteer Spotlight” Series last year to highlight the stories of some of the many selfless volunteers who play a major role in making our work possible. We are truly grateful for all of the love and heart-felt effort each of our volunteers puts into organizing awareness events, blood drives […]
