Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy

Study Summary

This is a single-center observational study evaluating the potential value of NfL as a biomarker for diagnosis, detection of disease onset, monitoring of disease progression, and treatment response in asymptomatic carriers of TTR variants and symptomatic hATTR amyloidosis patients with polyneuropathy.

Conditions

Hereditary Amyloidosis, Transthyretin-Related Asymptomatic Carrier State

Interventions / Drugs

Standard of Care

View on ClinicalTrials.gov ↗

Sponsor

ALNY

Alnylam Pharmaceuticals

RNAi Therapeutics

F28/100

Study Details

Enrollment500 participants

Primary Completion—

Lead SponsorAlnylam Pharmaceuticals

Data Retrieved2026-04-11 02:53:26.219234+00:00

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