Top sponsors: Children's Hospital of Philadelphia (8) · Centre Hospitalier Universitaire de Nice (6) · Biogen (4) · Columbia University (4) · Lexeo Therapeutics (3)

Narrower conditions

Active trials

• Friedreich Ataxia 35
• Mitochondrial Myopathies 26
• Optic Atrophy, Hereditary, Leber 12
• Leigh Disease 9
• VLCAD deficiency 6
• Optic Atrophy, Autosomal Dominant 5
• Pyruvate Dehydrogenase Complex Deficiency Disease 5
• Carbamoyl-Phosphate Synthase I Deficiency Disease 3
• Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation 3

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Data updated daily from ClinicalTrials.gov.