Regresa al Registro Simple

 
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; Resultados cardiovasculares; Resultados renales
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


Ficheros en el ítem

Thumbnail

Este ítem aparece en la(s) siguiente(s) colección(ones)

Regresa al Registro Simple