KL1333 Mitochondria medicin

elvira

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About KL1333

KL1333 is being developed towards a treatment for a subset of adult primary mitochondrial disease patients suffering from multiple debilitating symptoms, including chronic fatigue and myopathy. Diagnoses can include MELAS-MIDD and KSS-CPEO spectrum disorders as well as MERRF syndrome. The KL1333 compound is a potent modulator of the cellular levels of NAD+ and NADH, central co-enzymes in the cell’s energy metabolism. In a cohort of PMD patients in a Phase 1a/b study, the patients who received KL1333 showed both improvements in symptoms of fatigue as well as functional improvements. KL1333 has received orphan drug designation in both the USA and Europe.
 
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About KL1333

KL1333 is being developed towards a treatment for a subset of adult primary mitochondrial disease patients suffering from multiple debilitating symptoms, including chronic fatigue and myopathy. Diagnoses can include MELAS-MIDD and KSS-CPEO spectrum disorders as well as MERRF syndrome. The KL1333 compound is a potent modulator of the cellular levels of NAD+ and NADH, central co-enzymes in the cell’s energy metabolism. In a cohort of PMD patients in a Phase 1a/b study, the patients who received KL1333 showed both improvements in symptoms of fatigue as well as functional improvements. KL1333 has received orphan drug designation in both the USA and Europe.
The question is how it modulates NAD+/H. I think it increases it (I would like to know how?). If it does, there would be more electrons for the ATP synthesis. But the thing is: We don't actually know if that's the problem. Studies (some) are showing normal ATP levels.
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Depends if mitochondrial disease is truly at the heart of the illness. I guess the bigger question is which subset of patients may this improve? Although it would be great if it made 60% of patients better purely because they share a similar symptomatic overlap.