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How to START? Four pillars to optimally begin your orphan drug development

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 progression....

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Detalles Bibliográficos
Autores principales: Jonker, Anneliene Hechtelt, Batista, Liliana, Gabaldo, Michela, Hivert, Virginie, Ardigo, Diego
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10398909/
https://www.ncbi.nlm.nih.gov/pubmed/37537670
http://dx.doi.org/10.1186/s13023-023-02845-9