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dc.contributorVall d'Hebron Barcelona Hospital Campus
dc.contributor.authorBeck, Michael
dc.contributor.authorRamaswami, Uma
dc.contributor.authorHernberg‑Ståhl, Elizabeth
dc.contributor.authorHughes, Derralynn
dc.contributor.authorKampmann, Christoph
dc.contributor.authorMehta, Atul
dc.contributor.authorPintos Morell, Guillem
dc.date.accessioned2022-09-09T12:37:18Z
dc.date.available2022-09-09T12:37:18Z
dc.date.issued2022-06-20
dc.identifier.citationBeck M, Ramaswami U, Hernberg-Ståhl E, Hughes DA, Kampmann C, Mehta AB, et al. Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry. Orphanet J Rare Dis. 2022 Jun 20;17:238.
dc.identifier.issn1750-1172
dc.identifier.urihttp://hdl.handle.net/11351/8115
dc.descriptionAgalsidasa alfa; Resultats cardiovasculars; Resultats renals
dc.description.sponsorshipFOS is funded by Takeda Pharmaceuticals International AG, which also assisted in analyzing the data and preparing the manuscript. Takeda Development Center Americas, Inc. provided funding to Excel Medical Affairs for support in writing and editing this manuscript.
dc.language.isoeng
dc.publisherBMC
dc.relation.ispartofseriesOrphanet Journal of Rare Diseases;17
dc.rightsAttribution 4.0 International
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/
dc.sourceScientia
dc.subjectMetabolisme - Trastorns - Tractament
dc.subjectEnzims - Ús terapèutic
dc.subjectMalalties rares - Tractament
dc.subject.meshEnzyme Replacement Therapy
dc.subject.meshFabry Disease
dc.subject.mesh/drug therapy
dc.subject.meshRegistries
dc.titleTwenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry
dc.typeinfo:eu-repo/semantics/article
dc.identifier.doi10.1186/s13023-022-02392-9
dc.subject.decstratamiento de sustitución enzimática
dc.subject.decsenfermedad de Fabry
dc.subject.decs/farmacoterapia
dc.subject.decssistema de registros
dc.relation.publishversionhttps://doi.org/10.1186/s13023-022-02392-9
dc.type.versioninfo:eu-repo/semantics/publishedVersion
dc.audienceProfessionals
dc.contributor.organismesInstitut Català de la Salut
dc.contributor.authoraffiliation[Beck M] SphinCS GmbH, Institute Clinical Science LSD, Hochheim, Germany. [Ramaswami U, Hughes DA] Lysosomal Disorders Unit, Institute of Immunity and Transplantation, Royal Free London NHS Foundation Trust, University College London, London, UK. [Hernberg-Ståhl E] Late Phase Solutions Europe AB, Täby, Sweden. [Kampmann C] Johannes Gutenberg School of Medicine, University of Mainz, Mainz, Germany. [Mehta AB] Department of Haematology, University College London, London, UK. [Pintos-Morell G] Reference Centre for Hereditary Metabolic Disorders (MetabERN), Vall d’Hebron Hospital Universitari, Barcelona, Spain
dc.identifier.pmid35725623
dc.identifier.wos000813766600004
dc.rights.accessrightsinfo:eu-repo/semantics/openAccess


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