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...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2024-12-01
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| Series: | Pediatric Hematology Oncology Journal |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2468124524000615 |