Successes and pitfalls in orphan drug development for sickle cell disease
| Title: | Successes and pitfalls in orphan drug development for sickle cell disease |
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| Authors: | Costa, Enrico, Isgrò, Antonella, de Montalembert, Mariane, Leufkens, Hubert G. M., Ware, Russell E., De Franceschi, Lucia |
| Source: | In Blood Advances 28 May 2024 8(10):2455-2465 |
| Database: | ScienceDirect |
| ISSN: | 24739529 |
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| DOI: | 10.1182/bloodadvances.2023011730 |
| Published in: | Blood Advances |
| Language: | English |