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.
Authors
- Dianna Quan (ORCID: https://orcid.org/0000-0002-3699-5252)
- Miriam Freimer (ORCID: https://orcid.org/0000-0001-8282-764X)
- Brett W. Sperry (ORCID: https://orcid.org/0000-0002-8210-6401)
- Joel Fernandez (ORCID: https://orcid.org/0000-0003-4008-4301)
- Francy Shu (ORCID: https://orcid.org/0000-0002-4670-1561)
- Elena Yureneva
- Ugochukwu Egolum (ORCID: https://orcid.org/0000-0002-9606-1347)
- Saurabh Malhotra (ORCID: https://orcid.org/0000-0002-9396-9579)
- Steven Roblin
- Urvi Desai (ORCID: https://orcid.org/0000-0002-5169-3201)
- Karthikeyan Ananthasubramaniam (ORCID: https://orcid.org/0000-0001-5837-496X)
- Shaun Bender (ORCID: https://orcid.org/0000-0002-7589-4534)
- Kelley E. Capocelli (ORCID: https://orcid.org/0000-0002-8256-2896)
- Ronald Zolty (ORCID: https://orcid.org/0000-0003-1749-4046)
- Patrick Y. Jay (ORCID: https://orcid.org/0000-0001-9429-5492)
- Elizabeth A. Mauricio (ORCID: https://orcid.org/0000-0002-9493-9777)
- Roy S. Small
- Yessar Hussain
Institutions
- 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)
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
- 0.00