How to START? Four pillars to optimally begin your orphan drug development
Abstract Drug development is a complex, resource intensive and long process in any disease area, and developing medicines to treat rare diseases presents even more challenges due to the small patient populations, often limited disease knowledge, heterogeneous clinical manifestations, and disease pro...
Main Authors: | Anneliene Hechtelt Jonker, Liliana Batista, Michela Gabaldo, Virginie Hivert, Diego Ardigo, on behalf of the IRDiRC ODDG TF and IRDiRC TSC |
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Format: | Article |
Language: | English |
Published: |
BMC
2023-08-01
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Series: | Orphanet Journal of Rare Diseases |
Subjects: | |
Online Access: | https://doi.org/10.1186/s13023-023-02845-9 |
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