The KHENERGY Study: Safety and Efficacy of KH176 in Mitochondrial m.3243A>G Spectrum Disorders

KH176 is a potent intracellular reduction–oxidation‐modulating compound developed to treat mitochondrial disease. We studied tolerability, safety, pharmacokinetics, pharmacodynamics, and efficacy of twice daily oral 100 mg KH176 for 28 days in a double‐blind, randomized, placebo‐controlled, two‐way...

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Veröffentlicht in:	Clinical pharmacology and therapeutics 2019-01, Vol.105 (1), p.101-111
Hauptverfasser:	Janssen, Mirian C.H., Koene, Saskia, Laat, Paul, Hemelaar, Pleun, Pickkers, Peter, Spaans, Edwin, Beukema, Rypko, Beyrath, Julien, Groothuis, Jan, Verhaak, Chris, Smeitink, Jan
Format:	Artikel
Sprache:	eng
Schlagworte:	Administration, Oral Adult Antioxidants - administration & dosage Chromans - administration & dosage Cross-Over Studies DNA, Mitochondrial - genetics Double-Blind Method Female Humans Male Middle Aged Mitochondrial Diseases - diagnosis Mitochondrial Diseases - drug therapy Mitochondrial Diseases - genetics Mutation - genetics Treatment Outcome
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