New hope for rare muscle disease: experimental treatment aims to boost energy in cells
NCT ID NCT03639701
First seen Feb 02, 2026 · Last updated May 22, 2026 · Updated 20 times
Summary
This study tests two natural substances (deoxythymidine and deoxycytidine) as a treatment for TK2 deficiency, a rare genetic disorder that causes severe muscle weakness and breathing problems. The treatment is designed to help cells make more mitochondrial DNA, which may reduce symptoms. About 23 participants with confirmed TK2 deficiency will receive the treatment to see if it is safe and helpful.
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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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Contacts and locations
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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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