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; Resultados cardiovasculares; Resultados renales |
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 |