Thymidine Kinase Deficiency
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency [Phase 2]
An open-label Phase 2 study evaluates continuation treatment with a combination of pyrimidine nucleosides in patients with thymidine kinase …
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EMA approval · 2026-01-30First treatment for rare thymidine kinase 2 deficiency
The European Medicines Agency approved Kygevvi as the first treatment for thymidine kinase 2 deficiency, with reported improvements in motor…
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CLINICAL_TRIALS research · 2026-01-28Clinical Trial Update: Treatment of TK2 Deficiency With Thymidine and Deoxycytidine [Phase 1, Phase 2]
This clinical trial entry reports a Phase 1/2 study evaluating thymidine and deoxycytidine for TK2 deficiency. The trial is currently active…
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CLINICAL_TRIALS research · 2025-07-28Clinical Trial Update: Doxecitin and Doxribthymine in Adult Subjects With Thymidine Kinase 2 (TK2) Deficiency [Phase 2]
Phase 2 clinical trial update on Doxecitin and Doxribthymine for adults with thymidine kinase 2 deficiency–related mitochondrial myopathies,…
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CLINICAL_TRIALS withdrawal · 2023-09-01Clinical Trial Update: A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve) [Phase 3]
A Phase 3 trial evaluating MT1621 for Thymidine Kinase 2 deficiency was withdrawn.
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