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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...
Autores principales: | Nony, Patrice, Kurbatova, Polina, Bajard, Agathe, Malik, Salma, Castellan, Charlotte, Chabaud, Sylvie, Volpert, Vitaly, Eymard, Nathalie, Kassai, Behrouz, Cornu, Catherine |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2014
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Materias: | |
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 |
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