Final results of the ASCEND trial: Continued or sustained improvements in hepatosplenomegaly, respiratory outcomes, and lipid profile after 4 years of olipudase alfa enzyme replacement therapy in adults with acid sphingomyelinase deficiency

Basit Öğe Kaydı
dc.contributor.authorWasserstein, Melissa
dc.contributor.authorGallagher, Renata C.
dc.contributor.authorBarbato, Antonio
dc.contributor.authorGuigliani, Roberto
dc.contributor.authorGuelbert, Norberto
dc.contributor.authorHennermann, Julia B.
dc.contributor.authorIkezoe, Takayuki
dc.date.accessioned2025-11-16T19:33:51Z
dc.date.issued2025
dc.departmentİstanbul Medeniyet Üniversitesi
dc.description.abstract[Abstract Not Available]
dc.identifier.doi10.1016/j.ymgme.2024.108979
dc.identifier.issn1096-7192
dc.identifier.issn1096-7206
dc.identifier.issue2
dc.identifier.urihttps://doi.org/10.1016/j.ymgme.2024.108979
dc.identifier.urihttps://hdl.handle.net/20.500.14730/15166
dc.identifier.volume144
dc.identifier.wosWOS:001460878000010
dc.identifier.wosqualityN/A
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.titleFinal results of the ASCEND trial: Continued or sustained improvements in hepatosplenomegaly, respiratory outcomes, and lipid profile after 4 years of olipudase alfa enzyme replacement therapy in adults with acid sphingomyelinase deficiency
dc.typeConference Object

Dosyalar

Koleksiyon

WoS İndeksli Yayınlar Koleksiyonu