Duchenne Muscular Dystrophy (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.

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 Therapeutics, it is a one-time infusion that is FDA-approved for use in children ages four and five. Now, a new clinical trial begins at UC Davis Health for patients as young as two.

"John-John's Journey" chronicles one family's urgent trek across the globe to enroll their toddler in this gene replacement therapy clinical trial. 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. The new trial will administer a one-time infusion that will hopefully halt the progression of this deadly disease for John-John.

Read more about this story: health.ucdavis.edu/news/headlines/uc-davis-health-…
Duchenne Muscular Dystrophy clinical trials at UC Davis: clinicaltrials.ucdavis.edu/duchenne-muscular-dystr…
See the latest news from UC Davis Health: health.ucdavis.edu/newsroom

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0:00 Gene replacement therapy for DMD
2:01 What is Duchenne Muscular Dystrophy?
4:31 John-John's journey begins
7:24 Baseline appointment at UC Davis
9:23 Infusion day: Pharmacy prep
12:00 Infusion day: Treatment
14:47 6 months later
17:01 John-John's future

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Medical graphics provided by Serepta Therapeutics.

The information in this video was accurate as of the upload date, 12/19/24. For information purposes only. Consult your local medical authority for advice.

#dmd #musculardystrophy #genetherapy #sarepta #ucdavis ‪@MDA‬