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2:24
YouTube
NationwideChildrens
SMA Type 1: How Gene Therapy Works
Connect with a specialist: http://bit.ly/2nIxt7t Meet Jerry Mendell, MD: http://bit.ly/2nCirzT More about SMA Type 1: http://bit.ly/2nF1aWM More on Research: http://bit.ly/2nIy49d A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical ...
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