Principled compassion, 'right to try'

Principled compassion, 'right to try' . Editorial, Nature Medicine 20, 1355 (2014) doi:10.1038/nm.3772

As the momentum of right-to-try legislation demonstrates, the public increasingly demands access to the compassionate use of investigational treatments, and companies and regulators need to adapt to this new reality.

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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