A methodological framework for drug development in rare diseases

INTRODUCTION: Developing orphan drugs is challenging because of their severity and the requisite for effective drugs. The small number of patients does not allow conducting adequately powered randomized controlled trials (RCTs). There is a need to develop high quality, ethically investigated, and ap...

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Detalles Bibliográficos
Autores principales: Nony, Patrice, Kurbatova, Polina, Bajard, Agathe, Malik, Salma, Castellan, Charlotte, Chabaud, Sylvie, Volpert, Vitaly, Eymard, Nathalie, Kassai, Behrouz, Cornu, Catherine
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2014
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4255937/
https://www.ncbi.nlm.nih.gov/pubmed/25774598
http://dx.doi.org/10.1186/s13023-014-0164-y

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4255937/
https://www.ncbi.nlm.nih.gov/pubmed/25774598
http://dx.doi.org/10.1186/s13023-014-0164-y

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