A methodological framework for drug development in rare diseases

A methodological framework for drug development in rare diseases. Patrice Nony, Polina Kurbatova, Agathe Bajard, Salma Malik, Charlotte Castellan, Sylvie Chabaud, Vitaly Volpert, Nathalie Eymard, Behrouz Kassai, Catherine Cornu and The CRESim and Epi-CRESim study groups; Orphanet Journal of Rare Diseases 2014, 9:164 doi:10.1186/s13023-014-0164-y

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