Hope for rare muscle disease: experimental treatment trial advances
NCT ID NCT03845712
Summary
This study continues testing an experimental combination therapy (doxecitine and doxribtimine) in people with thymidine kinase 2 deficiency, a rare genetic disorder that causes muscle weakness and breathing problems. The research aims to see if long-term treatment is safe and whether it helps maintain or improve muscle function, breathing, and quality of life. It includes 47 participants who were already receiving similar treatments in a previous study.
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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
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