Leigh’s Disease is a rare, inherited neurodegenerative disorder caused by a defect in the function of mitochondria within the cells of the body. Mitochondria are responsible for creating more than 90% of the energy needed by the body to sustain life and support growth. When they fail, less and less energy is generated within the cells The infant first is unable to absorb food, and thus cannot grow and gain weight as expected (failure to thrive). Progressive damage to the central nervous system may lead to loss of mental and motor ability. There is no cure yet for Leigh’s Disease, but some experimental therapies are showing promise for treatment in some affected children.
Note: As science advances, we continue to experience a fast-evolving landscape of potential new therapies. The families portrayed here offer perspective on coping with rare and/or life-limiting conditions. They also each represent a particular moment in time.
If you are considering a clinical trial for your child, please also see the Courageous Parents Network Guided Pathway on Clinical Trials.