Matches in SemOpenAlex for { <https://semopenalex.org/work/W4309968143> ?p ?o ?g. }
Showing items 1 to 87 of
87
with 100 items per page.
- W4309968143 endingPage "198" @default.
- W4309968143 startingPage "187" @default.
- W4309968143 abstract "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 the first enzyme replacement therapy for non‐neurological symptoms of AM. Previously, a potential relationship was identified between three MAN2B1 genotype/subcellular localization subgroups (G1, G2, and G3) and AM disease severity. In VA‐treated patients with AM, it is unknown if a relationship exists between MAN2B1 genotype/subcellular localization subgroups, antidrug antibodies (ADAs), and infusion‐related reactions (IRRs). This pooled analysis evaluated data from 33 VA‐treated patients with AM to investigate this relationship. Overall, 10 patients were positive for ADAs, 4 of whom had treatment‐emergent ADAs (G1: 3/7 [43%]; G2: 1/17 [6%]; G3: 0/9). Treatment‐emergent ADA‐positive patients with relatively high titers ( n = 2; G1: 1012 U/ml and G2: 440 U/ml) experienced mild/moderate IRRs that were well‐managed; patients with lower titers ( n = 2) experienced no IRRs. Overall, changes from baseline in serum oligosaccharides and immunoglobulin G levels did not vary between ADA‐positive and ADA‐negative patients, suggesting a similar effect of VA treatment regardless of ADA status in most patients. Clinical outcomes (3MSCT and 6MWT) were also similar in most patients regardless of ADA status. While further studies are needed, these data suggest a relationship between MAN2B1 genotype/subcellular localization subgroups and ADA development, with G1 and G2 subgroups more likely to develop ADAs and IRRs. Regardless, this study suggests that ADAs have limited effect on the clinical impact of VA in most patients with AM." @default.
- W4309968143 created "2022-11-30" @default.
- W4309968143 creator A5018602374 @default.
- W4309968143 creator A5044578719 @default.
- W4309968143 creator A5061715984 @default.
- W4309968143 creator A5071260981 @default.
- W4309968143 creator A5089481507 @default.
- W4309968143 date "2022-11-25" @default.
- W4309968143 modified "2023-10-14" @default.
- W4309968143 title "Relationship between <scp><i>MAN2B1</i></scp> genotype/subcellular localization subgroups, antidrug antibody detection, and long‐term velmanase alfa treatment outcomes in patients with alpha‐mannosidosis" @default.
- W4309968143 cites W1570427382 @default.
- W4309968143 cites W1984182083 @default.
- W4309968143 cites W1989168301 @default.
- W4309968143 cites W2055262857 @default.
- W4309968143 cites W2169964507 @default.
- W4309968143 cites W2172232925 @default.
- W4309968143 cites W2800234963 @default.
- W4309968143 cites W2805967323 @default.
- W4309968143 cites W4250513280 @default.
- W4309968143 cites W4309968143 @default.
- W4309968143 doi "https://doi.org/10.1002/jmd2.12349" @default.
- W4309968143 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/36873087" @default.
- W4309968143 hasPublicationYear "2022" @default.
- W4309968143 type Work @default.
- W4309968143 citedByCount "2" @default.
- W4309968143 countsByYear W43099681432022 @default.
- W4309968143 countsByYear W43099681432023 @default.
- W4309968143 crossrefType "journal-article" @default.
- W4309968143 hasAuthorship W4309968143A5018602374 @default.
- W4309968143 hasAuthorship W4309968143A5044578719 @default.
- W4309968143 hasAuthorship W4309968143A5061715984 @default.
- W4309968143 hasAuthorship W4309968143A5071260981 @default.
- W4309968143 hasAuthorship W4309968143A5089481507 @default.
- W4309968143 hasBestOaLocation W43099681431 @default.
- W4309968143 hasConcept C104317684 @default.
- W4309968143 hasConcept C126322002 @default.
- W4309968143 hasConcept C135763542 @default.
- W4309968143 hasConcept C141071460 @default.
- W4309968143 hasConcept C159654299 @default.
- W4309968143 hasConcept C203014093 @default.
- W4309968143 hasConcept C2775944032 @default.
- W4309968143 hasConcept C2776879804 @default.
- W4309968143 hasConcept C32611913 @default.
- W4309968143 hasConcept C49453240 @default.
- W4309968143 hasConcept C54355233 @default.
- W4309968143 hasConcept C64943373 @default.
- W4309968143 hasConcept C71924100 @default.
- W4309968143 hasConcept C86803240 @default.
- W4309968143 hasConcept C90924648 @default.
- W4309968143 hasConceptScore W4309968143C104317684 @default.
- W4309968143 hasConceptScore W4309968143C126322002 @default.
- W4309968143 hasConceptScore W4309968143C135763542 @default.
- W4309968143 hasConceptScore W4309968143C141071460 @default.
- W4309968143 hasConceptScore W4309968143C159654299 @default.
- W4309968143 hasConceptScore W4309968143C203014093 @default.
- W4309968143 hasConceptScore W4309968143C2775944032 @default.
- W4309968143 hasConceptScore W4309968143C2776879804 @default.
- W4309968143 hasConceptScore W4309968143C32611913 @default.
- W4309968143 hasConceptScore W4309968143C49453240 @default.
- W4309968143 hasConceptScore W4309968143C54355233 @default.
- W4309968143 hasConceptScore W4309968143C64943373 @default.
- W4309968143 hasConceptScore W4309968143C71924100 @default.
- W4309968143 hasConceptScore W4309968143C86803240 @default.
- W4309968143 hasConceptScore W4309968143C90924648 @default.
- W4309968143 hasFunder F4320309888 @default.
- W4309968143 hasIssue "2" @default.
- W4309968143 hasLocation W43099681431 @default.
- W4309968143 hasLocation W43099681432 @default.
- W4309968143 hasLocation W43099681433 @default.
- W4309968143 hasOpenAccess W4309968143 @default.
- W4309968143 hasPrimaryLocation W43099681431 @default.
- W4309968143 hasRelatedWork W2009966535 @default.
- W4309968143 hasRelatedWork W2019988726 @default.
- W4309968143 hasRelatedWork W2082530227 @default.
- W4309968143 hasRelatedWork W2088063203 @default.
- W4309968143 hasRelatedWork W2126731990 @default.
- W4309968143 hasRelatedWork W2171277769 @default.
- W4309968143 hasRelatedWork W2334821950 @default.
- W4309968143 hasRelatedWork W2365431450 @default.
- W4309968143 hasRelatedWork W2367640290 @default.
- W4309968143 hasRelatedWork W2436880144 @default.
- W4309968143 hasVolume "64" @default.
- W4309968143 isParatext "false" @default.
- W4309968143 isRetracted "false" @default.
- W4309968143 workType "article" @default.