The film, titled “John-John’s Journey: Gene Replacement Therapy Offers New Hope for Duchenne Muscular Dystrophy (DMD),” follows Dino Kong and Emily Kuo as they relocate from Hsinchu, Taiwan, to enroll their son, John, in the trial at Sacramento-based UC Davis Health.
DMD is a severe genetic disorder that progressively weakens muscles, including the heart and lungs, often leading to life-threatening complications. Symptoms typically appear in boys by age 2, according to a Dec. 19 news release. The trial at UC Davis is testing Elevidys, a gene replacement therapy developed by Sarepta Therapeutics. Elevidys replaces the faulty gene responsible for DMD with a functional one.
The therapy, administered as a one-time infusion, has already received FDA approval for children aged 4 and 5. John, at just 2 years old, is among the youngest participants in the trial, which aims to expand the treatment’s use to younger patients. The trial is led by Craig McDonald, MD, chair of the Department of Physical Medicine and Rehabilitation at UC Davis Health.
The documentary was produced by Pamela Wu, director of news and media relations at UC Davis Health, and videographer Chris Nelson.
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