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.

Atıf İçin Kopyala

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

BMJ open, cilt.10, sa.10, 2020 (SCI-Expanded)

Yayın Türü: Makale / Tam Makale
Cilt numarası: 10 Sayı: 10
Basım Tarihi: 2020
Doi Numarası: 10.1136/bmjopen-2019-035182
Dergi Adı: BMJ open
Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, EMBASE, MEDLINE, Directory of Open Access Journals
Anahtar Kelimeler: genetics, cardiomyopathy, chronic renal failure, stroke medicine, ENZYME REPLACEMENT THERAPY, UNCERTAIN DIAGNOSIS, AGALSIDASE BETA, FEMALE-PATIENTS, CHILDREN, BIOMARKER
Gazi Üniversitesi Adresli: Evet

Özet

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.