Successes and pitfalls in orphan drug development for sickle cell disease
Enrico Costa(Brigham and Women's Hospital), Lucia De Franceschi(University of Verona), Mariane de Montalembert(Hôpital Necker-Enfants Malades), Hubert G. M. Leufkens(Utrecht University), Russell E. Ware(Cincinnati Children's Hospital Medical Center), Antonella Isgrò(Italian Medicines Agency)
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