Mitapivat represents a significant advance in thalassemia management by targeting erythrocyte metabolism and providing the first FDA‐approved oral treatment for anemia in adults with α or β thalassemia.
Fnu Zainab, Abdul Haseeb Hasan, Muhammad Hafi Abid et al.· Health Science Reports· 0 citations
The evidence demonstrates that advances in disease-modifying therapies and potentially curative strategies are transforming the management of sickle cell disease, reinforcing the need for long-term studies and public policies that expand access to innovative technologies.
Rosane Rezende de Souza Giuliani, Felipe Santos Teixeira Martiniano, Nicole Mioto Medeiros et al.· Advances in Hematology and O...· 0 citations
Background: β-Thalassemia and other β-hemoglobinopathies arise from defective β-globin production, and reactivating fetal hemoglobin (HbF) is a well-established strategy to ameliorate disease severity. Two principal gene-therapy approaches—erythroid-specific γ-globin gene addition and shRNA/shmiRNA-mediated knockdown o...
K. Paschoudi, Ninos-Ioannis Vasiloudis, Fotios Papadopoulos et al.· Thalassemia Reports· 0 citations
This review synthesizes contemporary breakthroughs in HSCT biology, technology, and clinical application, critically examines unresolved barriers, and highlights future directions, including Artificial Intelligence (AI)-driven predictive analytics, universal donor platforms, and equitable global access strategies.
M. Abah, Ovwigho Ugbede, O. C. et al.· DS Reviews of Research in Li...· 0 citations
Advances in β-thalassaemia treatment over the past decade are discussed, including newly approved and late-stage therapies and their future implications, and remaining research gaps are highlighted.
K. Musallam, Sujit Sheth, Franco Locatelli et al.· The Lancet Haematology· 2 citations
Sickle cell disease (SCD) is a monogenic β-globin disorder characterized by hemoglobin S polymerization, hemolysis, and vaso-occlusion, yet it exhibits remarkable clinical heterogeneity. Despite advances in supportive care and disease-modifying therapies, substantial variability in severity, treatment response, and lon...
Dina Tluli, Omar Tluli, Abdelrahman A. Karen et al.· Hemoglobin· 0 citations
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