Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative.

Hughes, Derralynn; Aguiar, Patricio; Deegan, Patrick; Ezgu, FATİH; Frustaci, Andrea; Lidove, Olivier; Linhart, Ales; Lubanda, Jean-Claude; Moon, James; Nicholls, Kathleen; Niu, Dau-Ming; Nowak, Albina; Ramaswami, Uma; Reisin, Ricardo; Rozenfeld, Paula; Schiffmann, Raphael; Svarstad, Einar; Thomas, Mark; Torra, Roser; Vujkovac, Bojan; Warnock, David; West, Michael; Johnson, Jack; Rolfe, Mark; Feriozzi, Sandro

doi:10.1136/bmjopen-2019-035182

Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative.

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Hughes D. A., Aguiar P., Deegan P. B., Ezgu F. S., Frustaci A., Lidove O., ...More

BMJ open, vol.10, no.10, 2020 (SCI-Expanded)

Publication Type: Article / Article
Volume: 10 Issue: 10
Publication Date: 2020
Doi Number: 10.1136/bmjopen-2019-035182
Journal Name: BMJ open
Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, EMBASE, MEDLINE, Directory of Open Access Journals
Keywords: genetics, cardiomyopathy, chronic renal failure, stroke medicine, ENZYME REPLACEMENT THERAPY, UNCERTAIN DIAGNOSIS, AGALSIDASE BETA, FEMALE-PATIENTS, CHILDREN, BIOMARKER
Gazi University Affiliated: Yes

Abstract

Objectives The PRoposing Early Disease Indicators for Clinical Tracking in Fabry Disease (PREDICT-FD) initiative aimed to reach consensus among a panel of global experts on early indicators of disease progression that may justify FD-specific treatment initiation.