New hope for rare muscle disease: expanded access opens doors
NCT ID NCT06590493
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 23 times
Summary
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 and are at risk of major disability or death. The goal is to provide treatment while the drugs are still being studied.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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