Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease

Malm D, Stensland HMFR, Edvardsen Ø, Nilssen Ø. The natural course and complications of alpha-mannosidosis—a retrospective and descriptive study. J Inherit Metab Dis. 2014;37:79–82.

Article 
PubMed 

Google Scholar 

Wolfe JH. Disorders of glycoprotein degradation: α-mannosidosis, β-mannosidosis, fucosidosis, sialidosis, and aspartylglycosaminuria. In: Rosenberg R, Pascual J (eds). Rosenberg’s Molecular and Genetic Basis of Neurological and Psychiatric Disease, vol. 1. Elsevier, 2025, pp…