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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Detalles Bibliográficos
Autores principales: Kaufmann, Petra, Pariser, Anne R., Austin, Christopher
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Position Statement
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6219030/
https://www.ncbi.nlm.nih.gov/pubmed/30400963
http://dx.doi.org/10.1186/s13023-018-0936-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6219030/
https://www.ncbi.nlm.nih.gov/pubmed/30400963
http://dx.doi.org/10.1186/s13023-018-0936-x

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