Thymine kinase 2 deficiency

Clinical trials for Thymine kinase 2 deficiency explained in plain language.

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Rare disease drug now available through expanded access

Disease control 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 muta…

Matched conditions: THYMINE KINASE 2 DEFICIENCY

Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control

Last updated Jun 27, 2026 13:03 UTC

Thymine kinase 2 deficiency

Get alerted when new Thymine kinase 2 deficiency trials appear

Rare disease drug now available through expanded access