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Gene, enzyme replacement improve mice's breathing, limb strength

Summary by Pompe Disease News
Gene and enzyme replacement therapies for Pompe disease improved breathing and limb strength in a mouse model of the disease, a study showed. The researchers said the improvement was associated with glycogen clearance in the tongue, the lungs, and the diaphragm, the main muscle involved in respiratory control. Glycogen is a stored form of glucose, or sugar — the body’s main source of energy — that accumulates to toxic levels in Pompe patients. B…
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Pompe Disease News broke the news in on Thursday, May 8, 2025.
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