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....

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Detalles Bibliográficos
Autores principales: Meena, Naresh Kumar, Ralston, Evelyn, Raben, Nina, Puertollano, Rosa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
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

Internet

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

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