Voxelotor (GBT440) in pediatric sickle cell disease: A review
Sickle cell disease (SCD) was first described in 1910 in African Americans, and the mutant hemoglobin S (HbS) was identified by electrophoresis in 1948. Sickle cell disease is the first genetic disease to be molecularly defined - a single point mutation in the β-globin gene (GAG→GTG) results in subs...
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Elsevier,
2024-12-01T00:00:00Z.
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