Drug development for exceptionally rare metabolic diseases: challenging but not impossible

Published 2013 View Full Article

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Title
Drug development for exceptionally rare metabolic diseases: challenging but not impossible
Authors
Keywords
Rare disease, Orphan medicinal product, Inherited metabolic disease, Prevalence, Preclinical proof of concept
Journal
Orphanet Journal of Rare Diseases
Volume 8, Issue 1, Pages 179
Publisher
Springer Nature
Online
2013-11-16
DOI
10.1186/1750-1172-8-179
References
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