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Article

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

Clinical pharmacology and therapeutics - 1 Jan 2019

Janssen Mirian C H, Koene Saskia, de Laat Paul, Hemelaar Pleun, Pickkers Peter, Spaans Edwin, Beukema Rypko, Beyrath Julien, Groothuis Jan, Verhaak Chris, Smeitink Jan

Abstract excerpt

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 crossover phase IIA study in 18 adult m.3243A>G patients without cardiovascular involvement. Efficacy parameters...

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Identifiers

PMID: 30058726
DOI: 10.1002/cpt.1197
PMCID: PMC6704357

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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