Matches in SemOpenAlex for { <https://semopenalex.org/work/W4280491874> ?p ?o ?g. }
Showing items 1 to 95 of
95
with 100 items per page.
- W4280491874 endingPage "1448" @default.
- W4280491874 startingPage "1437" @default.
- W4280491874 abstract "PurposeIntrathecal (IT) idursulfase-IT for the treatment of cognitive impairment is being investigated in pediatric patients with neuronopathic mucopolysaccharidosis II (MPS II) in addition to intravenous idursulfase. In this article, we report the findings for 54 months of idursulfase-IT treatment in an ongoing phase I/II extension trial (NCT01506141).MethodsA total of 15 male participants with neuronopathic MPS II (aged 3-11 years at enrollment) who were previously treated with intravenous idursulfase entered the extension study. Idursulfase-IT 10 mg or 30 mg was administered monthly via an IT drug delivery device or lumbar puncture, if indicated. The primary endpoint was safety and tolerability; secondary endpoints included pharmacokinetics, cerebrospinal fluid glycosaminoglycan levels, and cognitive function.ResultsIn total, 15 participants received a median (range) of 50 (18-55) idursulfase-IT doses. Idursulfase-IT was generally well tolerated; there were no life-threatening adverse events (AEs) or deaths. Most serious AEs were related to the IT drug delivery device; only 2 serious AEs were related solely to idursulfase-IT. After treatment with idursulfase-IT, cerebrospinal fluid glycosaminoglycans were decreased in all participants; these decreases were maintained. Cognitive function was stabilized in 3 of 4 testable participants at month 55.ConclusionThese long-term results support the clinical development of idursulfase-IT for patients with MPS II with cognitive impairment." @default.
- W4280491874 created "2022-05-22" @default.
- W4280491874 creator A5000620336 @default.
- W4280491874 creator A5013944605 @default.
- W4280491874 creator A5069233327 @default.
- W4280491874 creator A5085150364 @default.
- W4280491874 creator A5085315279 @default.
- W4280491874 creator A5087887507 @default.
- W4280491874 date "2022-07-01" @default.
- W4280491874 modified "2023-10-16" @default.
- W4280491874 title "Long-term open-label phase I/II extension study of intrathecal idursulfase-IT in the treatment of neuronopathic mucopolysaccharidosis II" @default.
- W4280491874 cites W1982093927 @default.
- W4280491874 cites W1989295722 @default.
- W4280491874 cites W1993027304 @default.
- W4280491874 cites W1997831257 @default.
- W4280491874 cites W2012973972 @default.
- W4280491874 cites W2020409398 @default.
- W4280491874 cites W2027030407 @default.
- W4280491874 cites W2027292988 @default.
- W4280491874 cites W2034981978 @default.
- W4280491874 cites W2049736899 @default.
- W4280491874 cites W2092327321 @default.
- W4280491874 cites W2100636864 @default.
- W4280491874 cites W2117030035 @default.
- W4280491874 cites W2117500144 @default.
- W4280491874 cites W2117675141 @default.
- W4280491874 cites W2137890611 @default.
- W4280491874 cites W2148045371 @default.
- W4280491874 cites W2745716847 @default.
- W4280491874 cites W2754304291 @default.
- W4280491874 cites W2761204945 @default.
- W4280491874 cites W2884044665 @default.
- W4280491874 cites W2966275708 @default.
- W4280491874 cites W2972531075 @default.
- W4280491874 cites W3028080341 @default.
- W4280491874 cites W3090225575 @default.
- W4280491874 cites W3133565909 @default.
- W4280491874 doi "https://doi.org/10.1016/j.gim.2022.04.002" @default.
- W4280491874 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/35588317" @default.
- W4280491874 hasPublicationYear "2022" @default.
- W4280491874 type Work @default.
- W4280491874 citedByCount "6" @default.
- W4280491874 countsByYear W42804918742022 @default.
- W4280491874 countsByYear W42804918742023 @default.
- W4280491874 crossrefType "journal-article" @default.
- W4280491874 hasAuthorship W4280491874A5000620336 @default.
- W4280491874 hasAuthorship W4280491874A5013944605 @default.
- W4280491874 hasAuthorship W4280491874A5069233327 @default.
- W4280491874 hasAuthorship W4280491874A5085150364 @default.
- W4280491874 hasAuthorship W4280491874A5085315279 @default.
- W4280491874 hasAuthorship W4280491874A5087887507 @default.
- W4280491874 hasBestOaLocation W42804918741 @default.
- W4280491874 hasConcept C126322002 @default.
- W4280491874 hasConcept C187212893 @default.
- W4280491874 hasConcept C197934379 @default.
- W4280491874 hasConcept C203092338 @default.
- W4280491874 hasConcept C2776225084 @default.
- W4280491874 hasConcept C2778375690 @default.
- W4280491874 hasConcept C2779134260 @default.
- W4280491874 hasConcept C2779969927 @default.
- W4280491874 hasConcept C2910638406 @default.
- W4280491874 hasConcept C535046627 @default.
- W4280491874 hasConcept C71924100 @default.
- W4280491874 hasConceptScore W4280491874C126322002 @default.
- W4280491874 hasConceptScore W4280491874C187212893 @default.
- W4280491874 hasConceptScore W4280491874C197934379 @default.
- W4280491874 hasConceptScore W4280491874C203092338 @default.
- W4280491874 hasConceptScore W4280491874C2776225084 @default.
- W4280491874 hasConceptScore W4280491874C2778375690 @default.
- W4280491874 hasConceptScore W4280491874C2779134260 @default.
- W4280491874 hasConceptScore W4280491874C2779969927 @default.
- W4280491874 hasConceptScore W4280491874C2910638406 @default.
- W4280491874 hasConceptScore W4280491874C535046627 @default.
- W4280491874 hasConceptScore W4280491874C71924100 @default.
- W4280491874 hasIssue "7" @default.
- W4280491874 hasLocation W42804918741 @default.
- W4280491874 hasLocation W42804918742 @default.
- W4280491874 hasOpenAccess W4280491874 @default.
- W4280491874 hasPrimaryLocation W42804918741 @default.
- W4280491874 hasRelatedWork W1699619364 @default.
- W4280491874 hasRelatedWork W1993518098 @default.
- W4280491874 hasRelatedWork W2117500144 @default.
- W4280491874 hasRelatedWork W2121504860 @default.
- W4280491874 hasRelatedWork W2143063409 @default.
- W4280491874 hasRelatedWork W2149588051 @default.
- W4280491874 hasRelatedWork W2153349311 @default.
- W4280491874 hasRelatedWork W2404010182 @default.
- W4280491874 hasRelatedWork W2913165266 @default.
- W4280491874 hasRelatedWork W4200610496 @default.
- W4280491874 hasVolume "24" @default.
- W4280491874 isParatext "false" @default.
- W4280491874 isRetracted "false" @default.
- W4280491874 workType "article" @default.