Motor Function, Cardiac Outcomes Improve With Gene Therapy in Pompe's Disease

(MedPage Today) — Children with infantile-onset Pompe’s disease experienced motor function and cardiac improvements after receiving an adeno-associated virus serotype 9 (AAV9)-mediated gene therapy, a pilot study showed.
Among four pediatric…

Source link : https://www.medpagetoday.com/neurology/generalneurology/116250

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Publish date : 2025-06-25 21:00:00

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Motor Function, Cardiac Outcomes Improve With Gene Therapy in Pompe’s Disease

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