Pioneering Oral Treatment for Thalassemia
B MC has played a key role in the groundbreaking ENERGIZE trial, the first study to demonstrate efficacy of mitapivat, an oral treatment for non-transfusion- dependent alpha- and beta-thalassemia. Led by Dr. Khaled Musallam as the Principal Investigator, BMC was a key contributor to the study. Later, the results of the ENERGIZE trial were presented at the plenary session of the 29th European Hematology Association Congress held in Madrid in June 2024. The results of the study will be used to file for a license from the FDA to use mitapivat as the first oral agent for treatment of non-transfusion- dependent thalassemia.
The results of the ENERGIZE trial were presented at the plenary session of the 29th European Hematology Association Congress held in Madrid in June 2024
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