1c051f718b9987a47d93d72c04c8025c pmc_10505750.pdf 31ba4daf24f107c8fb3e66593fddff06260ec232 pmc_10505750.pdf 2113cee7f9ddb1e6f92f75b10d8fc4f2308cadefe3c374724785bf823460bc7d pmc_10505750.pdf Title: Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study Subject: Objective: Fabry disease is a progressive disorder caused by deficiency of the α-galactosidase A enzyme (α-Gal A), leading to multisystemic organ damage with heterogenous clinical presentation. Author: Daniel G. Bichet; Robert J. Hopkin; Patrício Aguiar; Sridhar R. Allam; Yin-Hsiu Chien; Roberto Giugliani; Staci Kallish; Sabina Kineen; Olivier Lidove; Dau-Ming Niu; Iacopo Olivotto; Juan Politei; Paul Rakoski; Roser Torra; Camilla Tøndel; Derralynn A. Hughes Creator: Adobe InDesign 14.0 (Windows) Producer: Adobe PDF Library 15.0 CreationDate: Sat Aug 26 10:14:23 2023 CEST ModDate: Sat Aug 26 10:14:39 2023 CEST Custom Metadata: no Metadata Stream: yes Tagged: yes UserProperties: no Suspects: no Form: none JavaScript: no Pages: 19 Encrypted: no Page size: 595.276 x 841.89 pts (A4) Page rot: 0 File size: 2926145 bytes Optimized: no PDF version: 1.6 name type encoding emb sub uni object ID ------------------------------------ ----------------- ---------------- --- --- --- --------- XUAZZT+MuseoSans-500 Type 1C Custom yes yes yes 9 0 XUAZZT+MuseoSans-300 Type 1C Custom yes yes yes 11 0 XUAZZT+MuseoSans-700 Type 1C Custom yes yes yes 10 0 XUAZZT+STIXGeneral-Regular Type 1C Custom yes yes yes 12 0 OEKXDL+MuseoSans-300Italic Type 1C WinAnsi yes yes yes 275 0 MBLLBP+MinionPro-Regular Type 1C Custom yes yes yes 13 0 MBLLBP+MinionPro-It Type 1C Custom yes yes yes 14 0 MBLLBP+MuseoSans-700Italic Type 1C WinAnsi yes yes yes 118 0 MBLLBP+MinionPro-Bold Type 1C Custom yes yes yes 119 0 EPKPTF+MuseoSans-500Italic Type 1C WinAnsi yes yes yes 139 0 BIWXDL+STIXGeneral-Bold Type 1C Custom yes yes yes 238 0 BIWXDL+MinionPro-BoldIt Type 1C WinAnsi yes yes yes 239 0 Jhove (Rel. 1.28.0, 2023-05-18) Date: 2024-04-25 02:43:18 CEST RepresentationInformation: pmc_10505750.pdf ReportingModule: PDF-hul, Rel. 1.12.4 (2023-03-16) LastModified: 2024-04-24 02:39:24 CEST Size: 2926145 Format: PDF Version: 1.6 Status: Well-Formed and valid SignatureMatches: PDF-hul MIMEtype: application/pdf Profile: Tagged PDF PDFMetadata: Objects: 2819 FreeObjects: 1 IncrementalUpdates: 0 DocumentCatalog: ViewerPreferences: HideToolbar: false HideMenubar: false HideWindowUI: false FitWindow: false CenterWindow: false DisplayDocTitle: false NonFullScreenPageMode: UseNone Direction: L2R ViewArea: CropBox ViewClip: CropBox PrintArea: CropBox PageClip: CropBox PageLayout: SinglePage PageMode: UseNone Language: en-GB Outlines: Item: Title: Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study Children: Item: Title: 1. Introduction Item: Title: 2. Methods Children: Item: Title: 2.1. Selection of co-chairs and expert panel Item: Title: 2.2. Modified Delphi process Item: Title: 2.3. Statistical analyses Item: Title: 3. Results Item: Title: 4. Discussion Children: Item: Title: 4.1. α-Gal A measurements in patients receiving migalastat Item: Title: 4.2. Lyso-Gb3 Item: Title: 4.3. Recommended monitoring assessments Item: Title: 4.4. Treatment decisions Item: Title: 4.5. The consensus process in context Item: Title: 4.6. Strengths and limitations Item: Title: 4.7. Implications for future research Item: Title: 5. Conclusion Item: Title: Data availability statement Item: Title: Ethics statement Item: Title: Author contributions Item: Title: Funding Item: Title: Conflict of interest Item: Title: Publisher’s note Item: Title: Supplementary material Item: Title: References Info: Title: Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study Author: Daniel G. Bichet; Robert J. Hopkin; Patrício Aguiar; Sridhar R. Allam; Yin-Hsiu Chien; Roberto Giugliani; Staci Kallish; Sabina Kineen; Olivier Lidove; Dau-Ming Niu; Iacopo Olivotto; Juan Politei; Paul Rakoski; Roser Torra; Camilla Tøndel; Derralynn A. Hughes Subject: Objective: Fabry disease is a progressive disorder caused by deficiency of the α-galactosidase A enzyme (α-Gal A), leading to multisystemic organ damage with heterogenous clinical presentation. 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