Thymidine Kinase 2 Deficiency Drugs

Nov 10, 2025

In a significant milestone for the rare disease community, the FDA approved KYGEVVI (doxecitine and doxribtimine) in November 2025, marking the first and only approved treatment for thymidine kinase 2 deficiency (TK2d). This breakthrough approval represents a transformative moment for patients and families facing t...

Jun 09, 2025

Thymidine kinase 2 deficiency (TK2d) is a rare and life-threatening genetic condition and a type of mitochondrial disease. It stems from mutations in the TK2 gene, which is essential for the replication of mitochondrial DNA (mtDNA). These mutations lead to a depletion of mtDNA, resulting in profound muscle weakness...