Lifeline offered for rare genetic disorder as experimental drugs become available outside clinical trials
Disease control
AVAILABLE
This program provides early access to the investigational drugs doxecitine and doxribtimine for patients with Thymine Kinase 2 Deficiency (TK2d), a rare genetic disorder that causes muscle weakness and can be life-threatening. It's designed for patients who cannot participate in …
Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated Mar 25, 2026 14:09 UTC