Relationship between MAN2B1 genotype/subcellular localization subgroups, antidrug antibody detection, and long‐term velmanase alfa treatment outcomes in patients with alpha‐mannosidosis

Alpha‐mannosidosis (AM), an autosomal recessive disorder caused by pathogenic biallelic variants in the MAN2B1 gene, leads to lysosomal alpha‐mannosidase deficiency and accumulation of mannose‐rich oligosaccharides. Velmanase alfa (VA), a recombinant human lysosomal alpha‐mannosidase, is the first e...

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Detalles Bibliográficos
Autores principales: Borgwardt, Line Gutte, Ceravolo, Ferdinando, Zardi, Giulia, Ballabeni, Andrea, Lund, Allan Meldgaard
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Research Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9981422/
https://www.ncbi.nlm.nih.gov/pubmed/36873087
http://dx.doi.org/10.1002/jmd2.12349

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