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From scientific discovery to treatments for rare diseases – the view from the National Center for Advancing Translational Sciences – Office of Rare Diseases Research

We now live in a time of unprecedented opportunities to turn scientific discoveries into better treatments for the estimated 30 million people in the US living with rare diseases. Despite these scientific advances, more than 90% of rare diseases still lack an effective treatment. New data and geneti...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Kaufmann, Petra, Pariser, Anne R., Austin, Christopher
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6219030/
https://ncbi.nlm.nih.gov/pubmed/30400963
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0936-x
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