New hope for rare muscle disease: expanded access opens doors
Disease control
AVAILABLE
This program offers early access to two experimental drugs, doxecitine and doxribtimine, for people with thymine kinase 2 deficiency (TK2d), a rare genetic condition that causes severe muscle weakness. It is for pediatric and adult patients who have a confirmed TK2 gene mutation …
Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated May 17, 2026 17:19 UTC