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This Boy Struggled to Walk Before Gene Therapy. Now, Such Treatments Are Poised to Explode.

This Boy Struggled to Walk Before Gene Therapy. Now, Such Treatments Are Poised to Explode.

Conner Curran, now 10 years old, can walk more than two miles after gene therapy treatment for his Duchenne's muscular dystrophy.

Courtesy of the Curran family

Conner Curran was diagnosed with Duchenne's muscular dystrophy in 2015 when he was four years old. It's the most severe form of the genetic disease, with a nearly inevitable progression toward total paralysis. Many Duchenne's patients die in their teens; the average lifespan is 26.

But Conner, who is now 10, has experienced some astonishing improvements in recent years. He can now walk for more than two miles at a time – an impossible journey when he was younger.

In 2018, Conner became the very first patient to receive gene therapy specific to treating Duchenne's. In the initial clinical trial of nine children, nearly 80 percent reacted positively to the treatment). A larger-scale stage 3 clinical trial is currently underway, with initial results expected next year.

Gene therapy involves altering the genes in an individual's cells to stop or treat a disease. Such a procedure may be performed by adding new gene material to existing cells, or editing the defective genes to improve their functionality.

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Ron Shinkman
Ron Shinkman is a veteran journalist whose work has appeared in the New England Journal of Medicine publication Catalyst, California Health Report, Fierce Healthcare, and many other publications. He has been a finalist for the prestigious NIHCM Foundation print journalism award twice in the past five years. Shinkman also served as Los Angeles Bureau Chief for Modern Healthcare and as a staff reporter for the Los Angeles Business Journal. He has an M.A. in English from California State University and a B.A. in English from UCLA.
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