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Journal Articles

Successes and pitfalls in orphan drug development for sickle cell disease

Enrico Costa, Antonella Isgrò, Mariane de Montalembert, Hubert G. M. Leufkens, Russell E. Ware, Lucia De Franceschi
Journal: Blood Advances
Blood Adv (2024) 8 (10): 2455–2465.
https://doi.org/10.1182/bloodadvances.2023011730
Published: 2024
Includes: Supplemental data