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International collaborative actions and transparency to understand, diagnose, and develop therapies for rare diseases

Rare diseases, which affect over 350 million people worldwide and frequently go undiagnosed or misdiagnosed for years, suffer from sparse and dispersed medical knowledge leading to even rarer approved and effective therapeutic options for patients. A vast, unmet need for research and investment to a...

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Detalles Bibliográficos
Autores principales: Boycott, Kym M, Lau, Lilian PL, Cutillo, Christine M, Austin, Christopher P
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6505568/
https://www.ncbi.nlm.nih.gov/pubmed/30979709
http://dx.doi.org/10.15252/emmm.201910486