The emerging role of repurposed low-cost therapies, particularly hydroxyurea and thalidomide, as disease-modifying options in resource-limited settings are discussed and opportunities to democratise access to curative gene therapies through technological developments, decentralised manufacturing, regional collaboration...
Hanny T. Al-Samkari, F. Aboobacker, B. Andemariam et al.· The Lancet Haematology· 2 citations
A narrative review of emerging cancer treatment data, molecular profiling technologies, and existing clinicogenomic databases, focusing on how tumors are characterized, how subgroups are defined, and how demographic, lifestyle, and environmental factors are captured.
Farhood Farahnak, W. El-Deiry, Yves A. Lussier et al.· JCO Global Oncology· 0 citations
Several promising approaches targeting fetal hemoglobin induction, iron metabolism, and ineffective erythropoiesis have failed to demonstrate sufficient clinical benefit despite preclinical proof of concept, highlighting the complexity of therapeutic development in β-thalassemia.
K. M. Musallam, F. Locatelli, M. Algeri et al.· American journal of hematolo...· 0 citations
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