New hope for rare genetic disease: oral drug combo under study
NCT ID NCT03845712
First seen Feb 28, 2026 · Last updated May 02, 2026 · Updated 4 times
Summary
This study tests the safety and effectiveness of two oral drugs (doxecitine and doxribtimine) in people with thymidine kinase 2 (TK2) deficiency, a rare genetic condition that causes muscle weakness and breathing problems. About 47 participants who are already receiving similar treatment will continue their therapy under close monitoring. The goal is to see if the drugs are safe and help improve symptoms.
Disclaimer
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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.
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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Tk0102 1005
New York, New York, 10032, United States
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Tk0102 3031
Madrid, Spain
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Tk0102 3101
Seville, Spain
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Tk0102 3102
Barcelona, Spain
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Tk0102 3121
Esplugues de Llobregat, Spain
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Tk0102 4037
Nehariya, Israel
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Tk0102 4038
Haifa, Israel
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Tk0102 4039
Holon, Israel
Conditions
Explore the condition pages connected to this study.