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Drug Pipeline
RCT
Eplontersen reduces serum TTR in patients with hereditary transthyretin amyloidosis with polyneuropathy
Eplontersen Reduces Amyloid Protein in Hereditary Amyloidosis Patients
This exploratory analysis of the NEURO-TTRansform Phase 3 randomized trial evaluated eplontersen in patients with early- or late-onset ATTRv…
An exploratory analysis of a Phase 3 trial suggests eplontersen lowers amyloid protein levels and improves quality of life compared to histo…
Apr 24, 2026
Neurology
Cohort
Observational cohort finds high cardiac involvement and variable treatment patterns in hATTR patients.
Heart Treatment Often Misses Nerve Damage in This Group
This single-center retrospective cohort study of 54 patients with hereditary transthyretin amyloidosis (hATTR) found 94.4% had cardiac invol…
Heart patients with a common African American gene variant often get nerve damage missed, leaving tingling hands untreated while doctors foc…
medRxiv
Apr 19, 2026