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Patients with rare diseases: from therapeutic orphans to pioneers of personalized treatments

Common diseases, such as cancer, diabetes mellitus, or Alzheimer's disease, affect a large segment of the population, which justifies the enormous financial allocations for translational and clinical research. In contrast, the ~5,000 known rare disorders affect only very few patients each, even...

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Detalhes bibliográficos
Publicado no:EMBO Mol Med
Main Authors: Klein, Christoph, Gahl, William A
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5760852/
https://ncbi.nlm.nih.gov/pubmed/29180354
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201708365
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