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Hydroxyurea Dose Escalation Effective for Children with Sickle Cell Anemia

Extended follow-up of a phase 1/2 trial finds hydroxyurea dose optimization significantly improves clinical responses in children in sub-Saharan Africa with sickle cell.

Medical breakthrough: 8-year-old boy first in New York to receive FDA-approved gene therapy for rare inherited blood disorder

Medical breakthrough: 8-year-old boy first in New York to receive FDA-approved gene therapy for rare inherited blood disorder
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Boy with rare blood disorder receives new life-changing gene therapy treatment

Yusuf Saeed, an 8-year-old born with a rare genetic blood disorder called thalassemia, received a new and transformative gene therapy treatment at Cohen Children’s Medical Center on Long Island.

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