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Open access

Case report

First published 2024

Patisiran exposure in early pregnancy: a case report

Valentin Loser, Thomas Baumgartner, Hélène Legardeur [...]

Therapeutic Advances in Neurological Disorders

Open access

Review article

First published 2024

Efficacy and safety of patisiran for ATTRv-PN: a systematic review and meta-analysis

Xinyue Huang, Chong Sun, Haofeng Chen [...]

Therapeutic Advances in Neurological Disorders

Open access

Research article

First published 2025

Efficacy and safety of patisiran for the treatment of acquired amyloid polyneuropathy in domino liver transplant recipients

Velina Nedkova-Hristova, Laura Donadeu, Carmen Baliellas [...]

Therapeutic Advances in Neurological Disorders

Retricted access

Research article

First published 2020-06

Population Pharmacokinetic–Pharmacodynamic Model of Serum Transthyretin Following Patisiran Administration

Varun Goel, Nathalie H. Gosselin, Claudia Jomphe [...]

Nucleic Acid Therapeutics

Open access

Review article

First published 2023

A narrative review and expert recommendations on the assessment of the clinical manifestations,follow-up,and management of post-OLT patients with ATTRv amyloidosis

Carlos Casasnovas, Laura Lladó, Cristina Borrachero [...]

Therapeutic Advances in Neurological Disorders

Open access

Review article

First published 2025

Neurological efficacy and safety of RNA therapeutics in hereditary transthyretin amyloidosis: a systematic review and meta-analysis of randomized controlled trials

Maha Sajjad, Rabia Ashraf, Riya Bhagwan [...]

Therapeutic Advances in Cardiovascular Disease

Retricted access

Research article

First published 2026-2

Class-Specific Adverse Events of Patients Treated with Small Interfering RNA Therapeutics: A Disproportionality Analysis of the United States Food and Drug Administration Adverse Event Reporting System Database Based on the MY FAERS Platform

Ze Li, Xiaozhen Wang, Dandan Li [...]

Nucleic Acid Therapeutics

Open access

Research article

First published 2021-06

Pharmacodynamic and Pharmacokinetic Properties of Full Phosphorothioate Small Interfering RNAs for Gene Silencing In Vivo

Christian Berk, Gianluca Civenni, Yuluan Wang [...]

Nucleic Acid Therapeutics

Open access

Review article

First published 2021

Targeted Therapies for Hereditary Peripheral Neuropathies: Systematic Review and Steps Towards a ‘treatabolome’

Matthew J. Jennings, Angela Lochmüller, Antonio Atalaia [...]

Journal of Neuromuscular Diseases

Retricted access

Review article

First published 2021-12

Transthyretin Amyloid Cardiomyopathy—Current and Future Therapies

Jankhna D. Yadav, Harjot Othee, Kelly A. Chan [...]

Annals of Pharmacotherapy

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