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) identifier identifier identifier

  • 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


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.