doxribtimine

Doxribtimine is a synthetic pyrimidine deoxynucleoside, also known as deoxythymidine or thymidine. It is being developed as an oral therapy for thymidine kinase 2 deficiency (TK2d), a rare mitochondrial DNA depletion syndrome. Doxribtimine is typically administered in combination with doxecitine (deoxycytidine) as part of a fixed-dose nucleoside therapy regimen (MT1621). The mechanism involves supplementing deficient nucleosides to restore mitochondrial DNA replication fidelity and increase mtDNA copy number. This approach aims to compensate for impaired TK2 enzyme activity by providing substrates necessary for mtDNA synthesis and repair, thereby improving muscle function and survival in affected patients. Clinical studies have shown that this therapy can improve motor milestones and reduce mortality risk in TK2d patients[4][5][7][10].

Other names

deoxythymidinethymidine