Promising new therapy for rare mitochondrial disease enters human trials
NCT ID NCT03639701
First seen Feb 02, 2026 · Last updated May 13, 2026 · Updated 19 times
Summary
This study tests two natural substances, deoxythymidine and deoxycytidine, in 23 people with TK2 deficiency, a rare genetic condition that causes muscle weakness and breathing problems. The goal is to help cells make more mitochondrial DNA and reduce symptoms. The study focuses on safety and whether the treatment can improve survival and daily function.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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Columbia University Irving Medical Center
New York, New York, 10032, United States
Conditions
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