dc.contributor | Vall d'Hebron Barcelona Hospital Campus |
dc.contributor.author | Beck, Michael |
dc.contributor.author | Ramaswami, Uma |
dc.contributor.author | Hernberg‑Ståhl, Elizabeth |
dc.contributor.author | Hughes, Derralynn |
dc.contributor.author | Kampmann, Christoph |
dc.contributor.author | Mehta, Atul |
dc.contributor.author | Pintos Morell, Guillem |
dc.date.accessioned | 2022-09-09T12:37:18Z |
dc.date.available | 2022-09-09T12:37:18Z |
dc.date.issued | 2022-06-20 |
dc.identifier.citation | Beck 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.issn | 1750-1172 |
dc.identifier.uri | https://hdl.handle.net/11351/8115 |
dc.description | Agalsidasa alfa; Resultats cardiovasculars; Resultats renals |
dc.description.sponsorship | FOS 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.iso | eng |
dc.publisher | BMC |
dc.relation.ispartofseries | Orphanet Journal of Rare Diseases;17 |
dc.rights | Attribution 4.0 International |
dc.rights.uri | http://creativecommons.org/licenses/by/4.0/ |
dc.source | Scientia |
dc.subject | Metabolisme - Trastorns - Tractament |
dc.subject | Enzims - Ús terapèutic |
dc.subject | Malalties rares - Tractament |
dc.subject.mesh | Enzyme Replacement Therapy |
dc.subject.mesh | Fabry Disease |
dc.subject.mesh | /drug therapy |
dc.subject.mesh | Registries |
dc.title | Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry |
dc.type | info:eu-repo/semantics/article |
dc.identifier.doi | 10.1186/s13023-022-02392-9 |
dc.subject.decs | tratamiento de sustitución enzimática |
dc.subject.decs | enfermedad de Fabry |
dc.subject.decs | /farmacoterapia |
dc.subject.decs | sistema de registros |
dc.relation.publishversion | https://doi.org/10.1186/s13023-022-02392-9 |
dc.type.version | info:eu-repo/semantics/publishedVersion |
dc.audience | Professionals |
dc.contributor.organismes | Institut 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.pmid | 35725623 |
dc.identifier.wos | 000813766600004 |
dc.rights.accessrights | info:eu-repo/semantics/openAccess |