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Mitapivat Maintains Safety, Tolerability Over 2 Years in Sickle Cell Disease
Extension analysis from a phase 1 trial show the investigative oral mitapivat was not associated with any discontinuations nor deaths over 2 years.
San diego
United states
National institutes of health
Sickle cell branch of the national heart
Lung blood institute
American society of hematology
Swee lay thein
American society
National institutes
Sickle cell branch
National heart
Blood institute
Sickle cell disease
Sickle cell
Phase 1
Mash 2023
Mitapivat Effective, Safe in Treating SCD Through 1 Year
Durable mechanistic effects and a good safety profile were found in patients who took mitapivat for sickle cell disease (SCD).
Sickle cell
FDA Gave Approval to Oral Drug for Blood Cell Disorder
U.S. Food and Drug Administration approved Mitapivat for the treatment of adults with pyruvate kinase deficiency.
United states
Drug administration
Massachusetts general hospital
New england journal
Blood cell disorder
Pyruvate kinase deficiency
Genetic disorder
S fda
Ifelong anemia
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