KH176 under development for rare mitochondrial disease: a first in man randomized controlled clinical trial in healthy male volunteers

Abstract Background Mitochondrial disorders are a clinically, biochemically and genetically heterogeneous group of multi-system diseases, with an unmet medical need for treatment. KH176 is an orally bio-available small molecule under development for the treatment of mitochondrial(−related) diseases....

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Bibliographic Details
Main Authors: Saskia Koene, Edwin Spaans, Luc Van Bortel, Griet Van Lancker, Brant Delafontaine, Fabio Badilini, Julien Beyrath, Jan Smeitink
Format: Article
Language:English
Published: BMC 2017-10-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Online Access:http://link.springer.com/article/10.1186/s13023-017-0715-0