Lifeline offered for rare genetic disorder as experimental drugs become available outside clinical trials
NCT ID NCT06590493
Summary
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 regular clinical trials, particularly children with symptom onset before age 12. The program allows doctors to prescribe these medications while collecting safety and effectiveness data.
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Conditions
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