UC Davis Health premieres new documentary on Duchenne Muscular Dystrophy treatment

Today UC Davis Health premieres a new documentary on a family’s urgent journey across the globe to get their toddler in a clinical trial for patients with Duchenne Muscular Dystrophy.

“John-John's Journey: Gene Replacement Therapy Offers New Hope for Duchenne Muscular Dystrophy (DMD)” follows Dino Kong, Emily Kuo and their toddler son John. The family moves from Hsinchu, Taiwan, to Northern California, to enroll John in a UC Davis clinical trial for a promising therapy for DMD.

DMD is one of the most severe types of muscular dystrophy. The genetic disorder weakens all muscles in the body, including the heart and lungs. This can lead to heart and breathing problems, as well as early death. It affects mostly boys, who often show symptoms as early as age 2.

The drug Elevidys is seen as a breakthrough because it seeks to treat the root cause of DMD, and not just the symptoms. Manufactured by Sarepta, it replaces the faulty gene that causes DMD with a functional one. The one-time infusion is FDA-approved for use in children ages 4 and 5. Last August, UC Davis was the first health system in California to administer the treatment. At age 2, John is part of the latest trial to test the drug in younger kids. UC Davis is one of the primary sites for the Elevidys trial. It’s being led by Craig McDonald, professor and chair of the UC Davis Health Department of Physical Medicine and Rehabilitation.

“John-John's Journey” is produced by UC Davis Health Director of News and Media Relations Pamela Wu and Videographer Chris Nelson. Previous documentaries by the team of Wu and Nelson include “Separating Conjoined Twins at UC Davis Children’s Hospital” and “Searching for a Cure: The World's First Fetal Surgery Using Stem Cells for Spina Bifida.” The works are winners of the Northern California EMMY Award in the category of Health/Medical – Long-Form Content, respectively, in 2021 and 2023.