Rare disease drug now available through expanded access

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 mutation and are at risk of major disability or death. The goal is to offer treatment when no other options exist.