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- B-Cell Depletion and Immunomodulation Before Initiation of Enzyme Replacement Therapy Blocks the Immune Response To Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease Academic Article
- Clinical Outcomes After Long-Term Treatment With Alglucosidase Alfa in Infants and Children With Advanced Pompe Disease Academic Article
- Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II. Academic Article
- Mapping the T Helper Cell Response To Acid Alpha-Glucosidase in Pompe Mice Academic Article
- Neural Deficits Contribute To Respiratory Insufficiency in Pompe Disease Academic Article
- Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors. Academic Article
- Spinal Delivery of Aav Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice Academic Article