Fabry Disease Cardiac Biomarker: WORLDSymposium | Koneksa

Cardiac Biomarkers in Fabry Disease

Cardiac involvement is the leading cause of death in Fabry disease. However, no Fabry disease cardiac biomarker exists for monitoring it at home, and standard of care still addresses cardiac progression poorly. Patients feel that gap directly. In advisory sessions, they described not knowing when cardiac events occur, or how cardiac status shapes their daily fatigue.

This WORLDSymposium 2024 poster presents a composite measurement concept built to close it, developed by Koneksa with Amicus Therapeutics through a PRISMA literature search, nine patients living with Fabry disease, and eight treating clinicians.

View the poster and supplement, or contact us to discuss composite measurement design for your rare disease program.

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What You'll Learn

What patients ranked as most important, and the wearable, ECG, and diary burden they said they would accept

How the composite concept maps devices and instruments to cardiac, fatigue, functional, and renal domains

What clinicians prioritized for staging cardiac progression, including LVH boundaries and the KCCQ

Why the work aligns to the Evidentiary Framework (V3) and FDA digital health technology guidance

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