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Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease
Pompe disease, a deficiency of glycogen-degrading lysosomal acid alpha-glucosidase (GAA), is a disabling multisystemic illness that invariably affects skeletal muscle in all patients. The patients still carry a heavy burden of the disease, despite the currently available enzyme replacement therapy....
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2020
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7334420/ https://www.ncbi.nlm.nih.gov/pubmed/32671132 http://dx.doi.org/10.1016/j.omtm.2020.05.026 |