THYMIDINE KINASE 2 DEFICIENCY
Clinical trials for THYMIDINE KINASE 2 DEFICIENCY explained in plain language.
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Promising new therapy for rare mitochondrial disease enters human trials
Disease control OngoingThis 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 o…
Matched conditions: THYMIDINE KINASE 2 DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Columbia University • Aim: Disease control
Last updated May 13, 2026 16:03 UTC
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New hope for rare genetic disease: oral drug combo under study
Disease control OngoingThis 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 t…
Matched conditions: THYMIDINE KINASE 2 DEFICIENCY
Phase: PHASE2 • Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated May 04, 2026 16:18 UTC