Rare disease drug now available through expanded access
Disease control
AVAILABLE
This program provides access to two experimental drugs, doxecitine and doxribtimine, for people with thymine kinase 2 deficiency (TK2d), a rare genetic disorder that can cause severe muscle weakness and early death. It is for children and adults who have a confirmed TK2 gene muta…
Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:03 UTC