Tag: pharma
Tuesday, February 6th, 2024
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!! […]
Read more
: Blog, Care Walk, Events, Front Page, info foundation, info thalassemia, Links, Medical Research, News, person with thalassemia, Upcoming Events
: beta-thalassemia, Care Walk, care walk 2024, Care Walk Spotlight, CDC, Cooley's anemia, fundraiser, medical research, pharma, registration, Thal Pals, thalassemia, thalassemia awareness, thalassemia care, thalassemia care walk, thalassemia cure, thalassemia fundraiser, thalassemia medical research, what is thalassemia
Wednesday, January 17th, 2024
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 […]
Read more
: Blog, Front Page, info thalassemia, Links, Medical Research, News, person with thalassemia
: beta thalassemia cure, beta-thalassemia, casgevy, Cooley's anemia, CRISPR, gene editing, gene therapy, medical research, pharma, thalassemia, thalassemia cure, thalassemia treatment, vertex
Wednesday, January 3rd, 2024
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 […]
Read more
: Blog, Front Page, info thalassemia, medical professional, Medical Research, News, person with thalassemia, Webinars
: Agios, agospharmaceuticals, alpha thalassemia, beta-thalassemia, cooleys anemia foundation, energize, i have thalassemia, medical research for thalassemia, pharma, thalassemia, thalassemia care, thalassemia treatment, what is thalassemia
Tuesday, February 28th, 2023
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.
Read more
: Blog, Front Page, News, person with thalassemia
: Agios, beta thalassemia major, biotech, Cooley's anemia, e beta thalassemia, patient advocacy, pharma, PKD, Red Cell Revolution, SCD, Thal Pals, thalassemia
Friday, October 7th, 2022
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!
Read more
: Front Page, News, Webinars
: beta-thalassemia, bluebirdbio, gene therapy, pharma, Sujit Sheth, TDT, thalassemia, treatment, webinar, ZYNTEGLO
Thursday, September 22nd, 2022
The Eleventh Cooley’s Anemia Symposium will bring together basic scientists, clinical investigators, and clinicians to review and discuss recent research for thalassemia syndromes.
Read more
: Events, medical professional, Medical Research, Upcoming Events
: Cooley’s Anemia Symposium, medical research, New York Academy of Sciences, NYAS, Ongoing Clinical Research in Thalassemia, pharma
Wednesday, August 17th, 2022
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.
Read more
: Blog, Front Page, Medical Research, News
: beta-thalassemia, bluebird bio, Clinical Trials in Thalassemia Cell and Gene Therapy, Cooley's anemia, fda, gene therapy, pharma, thalassemia, ZYNTEGLO
Wednesday, March 31st, 2021
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 […]
Read more
: News
: Acceleron, pharma, scholarship
Monday, December 7th, 2020
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 […]
Read more
: News
: pharma
Sunday, December 6th, 2020
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 […]
Read more
: News
: pharma