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

Abstract 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 th...

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Hlavní autoři:	Line Gutte Borgwardt, Ferdinando Ceravolo, Giulia Zardi, Andrea Ballabeni, Allan Meldgaard Lund
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Wiley 2023-03-01
Edice:	JIMD Reports
Témata:	alpha‐mannosidosis antidrug antibody infusion‐related reactions MAN2B1 velmanase alfa
On-line přístup:	https://doi.org/10.1002/jmd2.12349
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