ECHS1 deficiency - Community for finding a cure - Gene therapy

Our mission is to fund ECHS1 gene therapy research at UT Southwestern Medical Center in Dallas. The pediatric neurology team is dedicated finding ...
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Other Rare Disease

  • Leigh Syndrome

Genetic Disorders

  • Inherited Metabolic Disorders

Mitochondrial Disorders

    If you've been diagnosed with any of these conditions, we're here to help.

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