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Doing it all - How Families are Reshaping Rare Disease Research

The face of rare disease drug discovery and development is changing right before our eyes. The outliers of the past were the plucky parents who summoned up the courage to try to treat their children against all odds. Think of the rare disease focused movies ‘Lorenzo’s Oil’ and ‘Extraordinary Measure...

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Detalhes bibliográficos
Publicado no:Pharm Res
Main Authors: Ekins, Sean, Perlstein, Ethan O.
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6159905/
https://ncbi.nlm.nih.gov/pubmed/30116974
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s11095-018-2481-7
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