THYMIDINE KINASE 2 DEFICIENCY
Clinical trials for THYMIDINE KINASE 2 DEFICIENCY explained in plain language.
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New hope for kids with rare muscle-wasting disease as drug combo moves to next phase
Disease control OngoingThis study tests two oral medications, doxecitine and doxribtimine, in 47 people with thymidine kinase 2 (TK2) deficiency, a rare genetic disorder that causes severe muscle weakness. Participants are already receiving similar treatment and will continue under close monitoring. Th…
Matched conditions: THYMIDINE KINASE 2 DEFICIENCY
Phase: PHASE2 • Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated May 26, 2026 09:30 UTC
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New hope for rare muscle disease: experimental treatment aims to boost energy in cells
Disease control OngoingThis 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 s…
Matched conditions: THYMIDINE KINASE 2 DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Columbia University • Aim: Disease control
Last updated May 22, 2026 13:54 UTC