Patients with rare diseases: from therapeutic orphans to pioneers of personalized treatments
Patients with rare diseases: from therapeutic orphans to pioneers of personalized treatments
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London: Nature Publishing Group UK
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English
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London: Nature Publishing Group UK
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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 though the cumulative disease burden is substantial. T...
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Patients with rare diseases: from therapeutic orphans to pioneers of personalized treatments
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TN_cdi_doaj_primary_oai_doaj_org_article_75237b6ae77e4877bc7558009543fa33
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_75237b6ae77e4877bc7558009543fa33
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ISSN
1757-4676
E-ISSN
1757-4684
DOI
10.15252/emmm.201708365
