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

Abstract 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 e...

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Bibliographic Details
Main Authors: Christoph Klein, William A Gahl
Format: Article
Language:English
Published: Springer Nature 2017-11-01
Series:EMBO Molecular Medicine
Online Access:https://doi.org/10.15252/emmm.201708365
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