The disease is heartbreaking. It turns babies into ragdolls and extinguishes lives just as they are getting started. But one USC Dornsife scientist is working to unravel the mystery behind the leading genetic cause of infant ...
Oct 13, 2011
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Researchers at the University of Missouri have identified a communication breakdown between nerves and muscles in mice that may provide new insight into the debilitating and fatal human disease known as spinal muscular atrophy ...
Sep 26, 2011
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Researchers in Ottawa report new hope for the treatment of infants born with serious genetic disorder.
Jul 25, 2011
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Dear Mayo Clinic: My husband and I want to expand our family and are trying to get pregnant with our first baby. I've read about family planning couples can do before conceiving and learned about carrier screening. We don't ...
Oct 19, 2023
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More than a billion people around the globe need assistive technology to go about everyday tasks independently. A new artificial intelligence (AI) wheelchair is taking assistive technology a step further and giving people ...
Jul 17, 2023
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New research published in Developmental Medicine & Child Neurology indicates that early identification and treatment of patients with spinal muscular atrophy (SMA)—a genetic disorder characterized by weakness and wasting ...
Jun 8, 2022
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The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in ...
Jul 2, 2020
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Swiss pharmaceuticals giant Novartis announced Monday it will buy US gene therapy company AveXis for $8.7 billion (7.1 billion euros).
Apr 9, 2018
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Zebrafish, roundworms, fruit flies, mice, rats, rabbits, dogs, cats, pigs, and monkeys provide steppingstones to clinical trials to evaluate new treatments for people. The value of animal studies continues, even after a new ...
Mar 30, 2018
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The Miami Project to Cure Paralysis, a Center of Excellence at the University of Miami Miller School of Medicine, in collaboration with Scholar Rock, a biotechnology company focused on discovering and developing drugs that ...
Jul 10, 2017
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