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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....
Autores principales: | Jonker, Anneliene Hechtelt, Batista, Liliana, Gabaldo, Michela, Hivert, Virginie, Ardigo, Diego |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2023
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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 |
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