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 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 […]
December 7, 2020 – Agios Pharmaceuticals reported preliminary findings from their Phase 2 study of mitapivat in non-transfusion-dependent thalassemia at the 2020 American Society of Hematology (ASH) virtual medical conference. Mitapivat activates the pyruvate kinase-R (PKR) enzyme, which improves red blood cell energy metabolism. Mitapivat was shown to improve ineffective erythropoiesis and anemia in a […]
December 6, 2020 – CRISPR Therapeutics and Vertex Pharmaceuticals have announced early results from their CLIMB Thal-111 Study in which transfusion-dependent beta thalassemia patients are treated with a gene editing therapy called CTX001. CTX001 is a therapy that involves CRISPR/Cas9 gene editing of a patient’s own hematopoietic stem cells (HSCs) in order to reactivate fetal […]
September 29, 2020 – Taro Pharmaceutical Industries Ltd. has launched specialty generic deferiprone (Ferriprox®) 500 mg tablets in the U.S. Further information can be found in the press release at: https://www.businesswire.com/news/home/20200928005649/en/Taro-Launches-Specialty-Generic-Deferiprone-Tablets-500mg-in-the-U.S At launch, Taro’s deferiprone tablets will be exclusively dispensed by BioPlus® Specialty Pharmacy. For more information, visit Taro Cares at www.tarocares.com or call 1-888-292-0744.
June 12, 2020 – CRISPR Therapeutics and Vertex Pharmaceuticals have announced early results from their CLIMB Thal-111 Study in which transfusion-dependent beta thalassemia patients are treated with a gene editing therapy called CTX001. CTX001 is a therapy that involves CRISPR/Cas9 gene editing of a patient’s own hematopoietic stem cells (HSCs) in order to reactivate fetal hemoglobin […]
June 12, 2020 – Agios Pharmaceuticals has provided an update on the preliminary findings from their Phase 2 study of mitapivat in non-transfusion-dependent thalassemia at the European Hematology Association (EHA) virtual medical congress. Mitapivat activates the pyruvate kinase-R enzyme, which improves red blood cell energy metabolism. Mitapivat was shown to improve ineffective erythropoiesis and anemia […]