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Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects - eBioMedicine
Immunomodulatory, liver depot gene therapy for Pompe disease - ScienceDirect
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Therapeutic efficacy of 3,4-Diaminopyridine phosphate on neuromuscular junction in Pompe disease - ScienceDirect
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Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease: Molecular Therapy - Methods & Clinical Development
Cardiovascular disease in non-classic Pompe disease: A systematic review - Neuromuscular Disorders
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