Patients with rare diseases: from therapeutic orphans to pioneers of personalized treatments
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DOI: 10.15252/emmm.201708365
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… read more here.
Keywords: medicine; orphans pioneers; rare diseases; therapeutic orphans ... See more keywords