Adults with chronic acid sphingomyelinase deficiency show significant visceral, pulmonary, and hematologic improvements after enzyme replacement therapy with olipudase-alfa: 1-year results of the ASCEND placebo-controlled trial

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dc.authorid0000-0002-4660-821X
dc.contributor.authorWasserstein, Melissa
dc.contributor.authorArash-Kaps, Laila
dc.contributor.authorBarbato, Antonio
dc.contributor.authorGallagher, Renata C.
dc.contributor.authorGiugliani, Roberto
dc.contributor.authorGuelbert, Norberto B.
dc.contributor.authorHollak, Carla
dc.date.accessioned2025-05-10T19:43:56Z
dc.date.issued2021
dc.departmentİstanbul Medeniyet Üniversitesi
dc.description17th Annual Research Meeting of the We're-Organizing-Research-for-Lysosomal-Diseases-Symposium (WORLDSymposium) -- FEB 07-12, 2021 -- ELECTR NETWORK
dc.description.abstract[No abstract available]
dc.description.sponsorshipWere Organizing Res Lysosomal Dis Symposium+
dc.identifier.doi10.1016/j.ymgme.2020.12.271
dc.identifier.endpageS110
dc.identifier.issn1096-7192
dc.identifier.issn1096-7206
dc.identifier.issue2
dc.identifier.scopusqualityQ2
dc.identifier.startpageS110
dc.identifier.urihttps://doi.org/10.1016/j.ymgme.2020.12.271
dc.identifier.urihttps://hdl.handle.net/20.500.14730/10779
dc.identifier.volume132
dc.identifier.wosWOS:000614651500267
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.titleAdults with chronic acid sphingomyelinase deficiency show significant visceral, pulmonary, and hematologic improvements after enzyme replacement therapy with olipudase-alfa: 1-year results of the ASCEND placebo-controlled trial
dc.typeConference Object

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