info thalassemia

U.S. Gene Therapy Trial for Thalassemia Receives FDA Approval

July17, 2012 Memorial Sloan-Kettering Cancer Center will begin evaluating a new stem-cell-based gene therapy for patients with the inherited blood disorder beta (β)-thalassemia. The clinical trial is the first to receive US Food and Drug Administration approval to treat this disease with genetically engineered cells and is a culmination of more than two decades of […]

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Questions on Thalassemia Syndromes: Intermedia and Hemoglobin H

February 1, 2012 – CAF Medical Advisory Board Chair Ellis Neufeld, MD, answers some of the questions which have been forwarded to CAF by individuals with thalassemia intermedia. CAF:  Why don’t intermedias qualify for bone marrow transplants, even if the diagnosis changes to major? EN:  Bone marrow transplantation can be a dangerous endeavor, and the […]

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