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Agios Pharmaceuticals to Present Broad Set of Clinical and Translational Data in Rare Blood ...

– Both Mitapivat Dose Arms Achieved Statistically Significant Hemoglobin Response in Phase 2 Portion of RISE UP Pivotal Study in Sickle Cell Disease, Compared to the Placebo Arm; Data Support ....

Krung Thep Mahanakhon , Rio De Janeiro , Estado Do Rio , San Diego , United States , Noord Brabant , New York , United Kingdom , Granda Ospedale Maggiore Policlinico , Angelo Dalessandro , Van Creveldkliniek , Bruno Fattizzo , Tarun Aurora , Thidarat Suksangpleng , Paola Bianchi , Charles Jonassaint , Biree Andemariam , Dan Budwick , Chris Taylor , Timothy Quang , Ariellel Langer , Modupe Idowu , Sujit Sheth , Jennifera Rothman , Andreas Glenth , Marissajm Traets ,

Beta-Thalassemia Gene Therapy Achieves Transfusion Independence

In patients with transfusion-dependent beta-thalassemia, a single gene therapy infusion is capable of yielding durable transfusion independence and substantial improvements in iron overload. ....

United States , Dana Farber Cancer Institute , Ariellel Langer , Alexisa Thompson , Ann Roberth Lurie , American Society Of Hematology , Drug Administration , Harvard Medical School , American Society , Genomic Medicine , Edicine Genomic , Beta Thalassemia , Gene Therapy , Precision Medicine , Hematologic Disorders , Iron Overload Disorder ,