Texas Boy Battles Rare Leigh's Disease

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New Hope for Leigh's Disease

Scientists are also working on a treatment that could help keep Will's mitochondria working indefinitely. In a small clinical trial, the experimental drug EPI-743 safely reversed some signs of Leigh's disease -- an exciting result that prompted orphan drug designation from the U.S. Food and Drug Administration. That means kids like Will can take the drug while further studies are carried out.

"We're pleased by the results for sure," said Dr. Guy Miller, chairman and CEO of Edison Pharmaceuticals, adding that a larger phase 2 trial of EPI-743 is set to start any day. "Our fingers are crossed."

Will has been taking EPI-743 for over a year, and his mom thinks it's helping. He has fewer falls, better bowel movements and has started to talk in sentences.

"We get hugs. We get to hear him say, 'I love you.' Some families don't get that," she said. "We feel like we're one of the luckiest unlucky families."

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