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Tags: Fadeel B

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A journey of hope: lessons learned from studies on rare diseases and orphan drugs

Rare diseases are frequently life-threatening or chronically debilitating and the impact on the quality of life of affected patients and their family members is thus significant. However, drug development for these conditions has been limited by a…

Tags: 2006, Backlog, Clinical Trials as Topic, Fadeel B, Hemophagocytic/drug therapy, Henter JI, Humans, Journal Article, Journal Of Internal Medicine, Lymphohistiocytosis, Orphan Drug Production/legislation & jurisprudence, Patient Advocacy, Rare Diseases/drug therapy, Severe Combined Immunodeficiency/drug therapy, Tyrosinemias/drug therapy, Wastfelt M

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