Tag: pharma

Agios Pharmaceuticals’ Oral Mitapivat Increases Hemoglobin Levels and Improves Markers of Erythropoiesis in Patients with Non-Transfusion-Dependent Thalassemia

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 […]

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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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Taro Pharmaceutical Launches Generic Deferiprone

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.

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Two Patients Become Transfusion Independent After Gene Editing Therapy

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 […]

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Agios Pharmaceuticals Provides Update on Phase 2 Study of Mitapivant in Non-Transfusion-Dependent Thalassemia

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 […]

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FDA Approves Twice-Daily Form of Ferriprox

A new formulation of the oral chelator Ferriprox, which is intended to be taken twice a day, has been approved for use in the United States by the U.S. Food and Drug Administration.  The previously approved formulation was to be taken three times daily; the new formulation eliminates the mid-day dose. “In the management of patients […]

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Navdeep Singh Selected for 2018 CAF-ApoPharma Distinguished Scholar Award

June 20, 2019 – The Cooley’s Anemia Foundation is proud to announce Navdeep Singh as the 2018 CAF-ApoPharma Distinguished Scholar Award recipient.  The award is in the amount of $20,000 and is presented to individuals with a clinically significant form of thalassemia who live in the United States and are pursuing postgraduate doctoral level studies […]

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ApoPharma Accepting Submissions for Iron Warriors Art Contest

September 25, 2018– ApoPharma welcomes submissions to its 15th Annual Iron Warriors Art Contest until the November 19, 2018 deadline. The goal of this contest is to raise awareness about thalassemia, to encourage the community to chelate, and to give the thalassemia community around the world the opportunity to share their artistic expression and inspire others. Check […]

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ApoPharma’s START Study Now Recruiting Participants

September 20, 2018 – The START study is examining the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on iron chelation therapy. There are 2 trial sites in the US: Los Angeles and Philadelphia. Learn […]

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Aaron Cheng Selected as 2017 CAF-ApoPharma Distinguished Scholar Award Recipient

March 13, 2018 – The Cooley’s Anemia Foundation is proud to announce Aaron Cheng as the 2017 CAF-ApoPharma Distinguished Scholar Award recipient. This scholarship is in the amount of $20,000 and is presented to individuals in the United States with a clinically significant form of thalassemia who are pursuing postgraduate doctoral level studies in medicine […]

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