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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This Jarvik-7 artificial heart was used in the first bridge operation in 1985 meant to replace a failing heart while the patient waited for a donor organ.

National Museum of American History

In June, a team of surgeons at Duke University Hospital implanted the latest model of an artificial heart in a 39-year-old man with severe heart failure, a condition in which the heart doesn't pump properly. The man's mechanical heart, made by French company Carmat, is a new generation artificial heart and the first of its kind to be transplanted in the United States. It connects to a portable external power supply and is designed to keep the patient alive until a replacement organ becomes available.

Many patients die while waiting for a heart transplant, but artificial hearts can bridge the gap. Though not a permanent solution for heart failure, artificial hearts have saved countless lives since their first implantation in 1982.

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Sarah Watts

Sarah Watts is a health and science writer based in Chicago. Follow her on Twitter at @swattswrites.

Elaine Kamil had just returned home after a few days of business meetings in 2013 when she started having chest pains. At first Kamil, then 66, wasn't worried—she had had some chest pain before and recently went to a cardiologist to do a stress test, which was normal.

"I can't be having a heart attack because I just got checked," she thought, attributing the discomfort to stress and high demands of her job. A pediatric nephrologist at Cedars-Sinai Hospital in Los Angeles, she takes care of critically ill children who are on dialysis or are kidney transplant patients. Supporting families through difficult times and answering calls at odd hours is part of her daily routine, and often leaves her exhausted.

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Lina Zeldovich
Lina Zeldovich has written about science, medicine and technology for Scientific American, Reader’s Digest, Mosaic Science and other publications. She’s an alumna of Columbia University School of Journalism and the author of the upcoming book, The Other Dark Matter: The Science and Business of Turning Waste into Wealth, from Chicago University Press. You can find her on http://linazeldovich.com/ and @linazeldovich.