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Curative Therapies for Hemophilias and Hemoglobinopathies in Adults: Immune, Gene, and Stem Cell Approaches in a Global Context

19 Aug 2025

Abstract excerpt

Hemophilias and hemoglobinopathies-including hemophilias A and B, sickle cell disease (SCD), and β-thalassemia-are debilitating genetic disorders associated with significant global health burdens. While traditional management has centered on factor replacement and transfusions, these approaches remain palliative, with limited access and durability in many regions. Recent advances in immune-based therapeutics...

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PMID: 40868273
DOI: 10.3390/biomedicines13082022

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