Lifeline extended: patients get continued access to mitochondrial disease drug
NCT ID NCT07159139
Summary
This program provides continued access to the drug vatiquinone for patients with inherited mitochondrial diseases, such as Leigh or Alpers syndrome. It is specifically for people who previously took the drug in a related clinical trial and, in their doctor's opinion, would benefit from staying on it. The goal is to allow continued treatment for those who cannot join another formal study.
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Conditions
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