MITOCHONDRIAL DISEASE
Clinical trials for MITOCHONDRIAL DISEASE explained in plain language.
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Brain-Boosting antioxidant tested for rare genetic disorder
Disease control Recruiting nowThis is an early safety study testing different doses of an antioxidant called N-acetylcysteine (NAC) in adults with a specific genetic mitochondrial disorder. The main goal is to find the highest safe dose that might help increase protective levels in the brain. Researchers will…
Matched conditions: MITOCHONDRIAL DISEASE
Phase: PHASE1 • Sponsor: Michio Hirano, MD • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC
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Lifeline extended: patients get continued access to mitochondrial disease drug
Disease control AVAILABLEThis 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 benefi…
Matched conditions: MITOCHONDRIAL DISEASE
Sponsor: Medical University of South Carolina • Aim: Disease control
Last updated Mar 20, 2026 14:47 UTC
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Tiny sensor seeks to unlock mysteries of devastating muscle diseases
Knowledge-focused Recruiting nowThis study is testing a new, minimally invasive sensor designed to measure oxygen levels in muscle tissue. Researchers want to see if this sensor can reliably detect differences between people with mitochondrial myopathy and healthy volunteers. The goal is to create a better tool…
Matched conditions: MITOCHONDRIAL DISEASE
Phase: PHASE1 • Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Mar 24, 2026 12:02 UTC