Impact of patisiran on polyneuropathy of hereditary transthyretin amyloidosis in patients with a V122I or T60A variant: a phase IV multicenter study

Patisiran demonstrated a consistent positive effect across multiple endpoints in patients with V122I/T60A ATTRv amyloidosis, including polyneuropathy manifestations.

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Publication Details

Journal
Annals of Medicine
Published
2025-09-20
DOI
https://doi.org/10.1080/07853890.2025.2537347
Primary Topic
Amyloidosis: Diagnosis, Treatment, Outcomes
Type
article
Field-Weighted Citation Impact
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article

Impact of patisiran on polyneuropathy of hereditary transthyretin amyloidosis in patients with a V122I or T60A variant: a phase IV multicenter study

Dianna Quan, Miriam Freimer, Brett W. Sperry, Joel Fernandez et al.
Annals of Medicine
Amyloidosis: Diagnosis, Treatment, Outcomes
article

Impact of patisiran on polyneuropathy of hereditary transthyretin amyloidosis in patients with a V122I or T60A variant: a phase IV multicenter study

Dianna Quan, Miriam Freimer, Brett W. Sperry, Joel Fernandez, Francy Shu, Elena Yureneva, Ugochukwu Egolum, Saurabh Malhotra, Steven Roblin, Urvi Desai, Karthikeyan Ananthasubramaniam, Shaun Bender, Kelley E. Capocelli, Ronald Zolty, Patrick Y. Jay, Elizabeth A. Mauricio, Roy S. Small, Yessar Hussain
article en

Abstract

Patisiran demonstrated a consistent positive effect across multiple endpoints in patients with V122I/T60A ATTRv amyloidosis, including polyneuropathy manifestations.

Annals of MedicineVol. 57(1)
Saint Luke's Health System (US), Alnylam Pharmaceuticals (United States) (US), University of South Florida (US), Baylor University Medical Center (US), Lancaster General Hospital (US), The Ohio State University Wexner Medical Center (US), Henry Ford Hospital (US), Northeast Georgia Medical Center (US), Mayo Clinic in Arizona (US), Saint Luke's Hospital (US), Baylor Scott & White Health (US), Wake Forest University (US), University of Colorado Anschutz Medical Campus (US), University of Nebraska Medical Center (US), Cook Medical (Denmark) (DK)
Openalex Percentile: Top 77%
Amyloidosis: Diagnosis, Treatment, Outcomes
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