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Pipeline

HK-1 Series
Symptoms in Mitochondrial Diseases

<Symptoms in Mitochondrial Diseases>

HK-1 Series

·Target: NADH:quinone oxidoreductase 1 (NQO1)
·Developmental stage: Non-clinical GLP toxicity study in progress
·Indication: Primary Mitochondrial Diseases (PMD)
Mitochondrial disease are a clinically heterogeneous group of disorders affecting the energy-converting process in mitochondria. Mitochondrial diseases are caused by mutation of genes encoded by either nuclear DNA or mitochondrial DNA. They can present at any age with almost any tissue and organs. The symptoms of mitochondrial diseases are extreme variability. The representative mitochondrial diseases are Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS), Leigh syndrome, Leber Hereditary Optic Neuropathy (LHON) and Myoclonic Epilepsy with Ragged Red Fiber (MERRF) syndrome. Supplements such as CoQ10, vitamins and nutrients are frequently used to treat mitochondrial diseases, but no treatment with proven efficacy is currently available.