hydroxyurea - Generic mfg.
Oxbryta (voxelotor) - Pfizer
Adakveo (crizanlizumab-tmca) - Novartis
Precision Medicine in Sickle Cell Disease: From Molecular Pathways to Curative Strategies. (PubMed, Hemoglobin) - Oct 16, 2026 - "Key pathogenic pathways including hemoglobin polymerization, vascular adhesion, and inflammatory and oxidative amplification have enabled the development of targeted agents such as hydroxyurea, voxelotor, crizanlizumab, and L-glutamine, each demonstrating domain-specific targets. Collectively, these advances support a paradigm shift from generalized symptom management toward biologically informed, individualized care. Continued integration of genomic profiling, biomarker stratification, and equitable implementation frameworks will be essential to translate precision strategies into sustainable improvements in global SCD outcomes." 
Journal • Review • Bone Marrow Transplantation • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation
http://www.ncbi.nlm.nih.gov/pubmed/42876272
 
Abdelrahman A Karen; Alaa Abdelhamid; Dahab Ghith; Dina Tluli; Mahd M Abujbara; Mohamed A Yassin; Omar Sorour; Omar Tluli
 
Oct 16, 2026
 
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