News

Story on Nicholas Jannetti Featured in Italian America Magazine

April 30, 2021 – A big thank you to our incredible friends at Order Sons and Daughters of Italy in America (OSDIA) for the beautiful story in the current Italian America Magazine on Nicholas Jannetti.  Nicholas was born with thalassemia but recently underwent a curative transplantation  and the article talks about his and his family’s […]

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Save the Date: CAF Patient-Family Conference on August 13-25

April 15, 2020 – CAF is enormously pleased to announce that this year’s Patient-Family Conference will be available as both an in-person and online event this year and will be held from August 13-15. The in-person event will be held in New Brunswick, NJ. However, please note that the number of people who can attend […]

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New CAF-Acceleron Scholarships for Academic Achievements Accepting Applications from U.S. Thalassemia Community

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 […]

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HealthWell Foundation Launches Pulmonary Hypertension Fund

January 11, 2021 – The HealthWell Foundation is an independent non-profit that provides a financial lifeline for inadequately insured Americans. They have launched a Pulmonary Hypertension Fund to assist individuals living with pulmonary hypertension (PH). Through the fund, HealthWell will provide up to $10,000 in medication copayment or insurance premium assistance for a 12-month grant period to eligible Medicare patients […]

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ASH Report: Luspatercept and Iron

December 6, 2020 – The effect of long-term luspatercept use on iron levels and on utilization of iron chelation therapy was assessed in the Phase 3 BELIEVE trial of luspatercept in adult patients with transfusion-dependent beta thalassemia. After 24 weeks of treatment, 17.0% of patients who took luspatercept showed a reduction in serum ferritin levels […]

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ASH Report: Early Results from CTX001 Gene Editing Study

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 […]

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ASH Report: Pediatric Results in bluebird bio Gene Therapy Trial

December 6, 2020 – Initial results from Phase 3 clinical trials showed that 10/12 adult patients with transfusion-dependent beta thalassemia achieved transfusion independence following treatment with bluebird bio’s beti-cel (LentiGlobin) gene therapy for β-thalassemia. After showing initial success in adult patients, these trials were expanded to include adolescents and children. Preliminary findings from the pediatric […]

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ASH Report: Long-Term Follow-Up Analysis of bluebird bio Gene Therapy

December 6, 2020 – Patients with transfusion-dependent beta thalassemia who were treated in bluebird bio’s clinical trials with beti-cel (LentiGlobin) gene therapy have shown sustained efficacy for up to six years in follow-up analysis. These findings were presented by Dr. Janet Kwiatkowski of the Children’s Hospital of Philadelphia at the 2020 American Society of Hematology […]

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