Continued improvement in adults with acid sphingomyelinase deficiency after 2 years of olipudase alfa in the ASCEND placebo-controlled trial

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dc.authorid0000-0002-4660-821X
dc.contributor.authorWasserstein, Melissa
dc.contributor.authorBarbato, Antonio
dc.contributor.authorGallagher, Renata
dc.contributor.authorGiugliani, Roberto
dc.contributor.authorGuelbert, Norberto
dc.contributor.authorHennermann, Julia
dc.contributor.authorHollak, Carla
dc.date.accessioned2025-05-10T19:49:33Z
dc.date.issued2022
dc.departmentİstanbul Medeniyet Üniversitesi
dc.descriptionACMG Annual Clinical Genetics Meeting -- MAR 22-26, 2022 -- Nashville, TN
dc.description.abstract[No abstract available]
dc.description.sponsorshipACMG
dc.identifier.doi10.1016/j.gim.2022.01.315
dc.identifier.endpageS177
dc.identifier.issn1098-3600
dc.identifier.issn1530-0366
dc.identifier.issue3
dc.identifier.scopusqualityQ1
dc.identifier.startpageS176
dc.identifier.urihttps://doi.org/10.1016/j.gim.2022.01.315
dc.identifier.urihttps://hdl.handle.net/20.500.14730/12075
dc.identifier.volume24
dc.identifier.wosWOS:000796586200066
dc.identifier.wosqualityQ1
dc.indekslendigikaynakWeb of Science
dc.language.isoen
dc.publisherElsevier Science Inc
dc.relation.ispartofGenetics in Medicine
dc.relation.publicationcategoryKonferans Öğesi - Uluslararası - Kurum Öğretim Elemanı
dc.rightsinfo:eu-repo/semantics/openAccess
dc.snmzKA_WOS_20250302
dc.titleContinued improvement in adults with acid sphingomyelinase deficiency after 2 years of olipudase alfa in the ASCEND placebo-controlled trial
dc.typeConference Object

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