CDC’s Division of Blood Disorders (DBD) is proud to offer its Public Health Webinar Series on Blood Disorders. The purpose of this series is to provide evidence-based information on new research, interventions, emerging issues of interest in blood disorders, as well as innovative approaches in collaborations and partnerships. We invite you to join us in this series.
Archives for 2020
Sujit Sheth, MD
Harold Weill Professor, Chief of Pediatric Hematology and
Oncology, and Vice Chair for Clinical Research, Department of
Pediatrics, Weill Cornell Medicine
Director, New York Comprehensive Thalassemia Center, New York-Presbyterian Hospital/Weill Cornell Medical Center
Overview of Novel Thalassemia Treatments
The thalassemia syndromes are among the most common genetic disorders. Homozygous or compound heterozygotes have a wide spectrum of clinical severity, with the most severe requiring regular blood transfusions to maintain health and prevent morbidity. Transfusion-associated iron overload and its complications result in significant morbidity and mortality, thus disease-modifying treatments are a major unmet need in the management of these individuals.
During the past five years, novel approaches to treatment have been possible with advances in technology. In this webinar, Dr. Sheth will discuss the basic pathophysiology of the disease and specific targets for novel therapies. He will review data from trials of the newer agents aimed at reducing or abolishing the transfusion burden and mention additional strategies that are in early stage development that have the potential to completely change the landscape of thalassemia care.
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