Two-year results of the ASCEND trial of olipudase alfa adults with chronic acid sphingomyelinase deficiency show parallel improvements in former placebo patients and further improvement in continuing olipudase alfa patients

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dc.authorid0000-0002-4660-821X
dc.contributor.authorWasserstein, Melissa P.
dc.contributor.authorBarbato, Antonio
dc.contributor.authorGallagher, Renata C.
dc.contributor.authorGiugliani, Roberto
dc.contributor.authorGuelbert, Norberto B.
dc.contributor.authorHennermann, Julia B.
dc.contributor.authorHollak, Carla
dc.date.accessioned2025-05-10T19:43:56Z
dc.date.issued2022
dc.departmentİstanbul Medeniyet Üniversitesi
dc.description18th Annual WORLD Symposium -- FEB 07-11, 2022 -- San Diego, CA
dc.description.abstract[No abstract available]
dc.identifier.doi10.1016/j.ymgme.2021.11.337
dc.identifier.endpageS127
dc.identifier.issn1096-7192
dc.identifier.issn1096-7206
dc.identifier.issue2
dc.identifier.scopusqualityQ2
dc.identifier.startpageS126
dc.identifier.urihttps://doi.org/10.1016/j.ymgme.2021.11.337
dc.identifier.urihttps://hdl.handle.net/20.500.14730/10780
dc.identifier.volume135
dc.identifier.wosWOS:000803066100324
dc.identifier.wosqualityQ2
dc.indekslendigikaynakWeb of Science
dc.language.isoen
dc.publisherAcademic Press Inc Elsevier Science
dc.relation.ispartofMolecular Genetics and Metabolism
dc.relation.publicationcategoryKonferans Öğesi - Uluslararası - Kurum Öğretim Elemanı
dc.rightsinfo:eu-repo/semantics/closedAccess
dc.snmzKA_WOS_20250302
dc.titleTwo-year results of the ASCEND trial of olipudase alfa adults with chronic acid sphingomyelinase deficiency show parallel improvements in former placebo patients and further improvement in continuing olipudase alfa patients
dc.typeConference Object

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