AAV-mediated delivery of secreted acid α-glucosidase with enhanced uptake corrects neuromuscular pathology in Pompe mice
Author:
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Link
https://insight.jci.org/articles/view/170199/files/pdf
Reference87 articles.
1. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease
2. Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study
3. Recombinant human acid -glucosidase: Major clinical benefits in infantile-onset Pompe disease
4. Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease
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