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Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.
JAMA Neurol
January 2025
Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy.
Importance: There is a lack of long-term efficacy and safety data on hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) and on RNA interference (RNAi) therapeutics in general. This study presents the longest-term data to date on patisiran for hATTR-PN.
Objective: To present the long-term efficacy and safety of patisiran in adults with hATTR-PN.
Solution biophysics identifies lipid nanoparticle non-sphericity, polydispersity, and dependence on internal ordering for efficacious mRNA delivery.
bioRxiv
December 2024
Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104, USA.
Lipid nanoparticles (LNPs) are the most advanced delivery system currently available for RNA therapeutics. Their development has accelerated since the success of Patisiran, the first siRNA-LNP therapeutic, and the mRNA vaccines that emerged during the COVID-19 pandemic. Designing LNPs with specific targeting, high potency, and minimal side effects is crucial for their successful clinical use.
View Article and Find Full Text PDFUse of technetium-99m-pyrophosphate single-photon emission computed tomography/computed tomography in monitoring therapeutic changes of RNA interference therapeutics in patients with hereditary transthyretin amyloid cardiomyopathy.
J Formos Med Assoc
October 2024
Department of Internal Medicine, Division of Cardiology, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan.
Article Synopsis
- RNA interference (RNAi) therapeutics, specifically patisiran and vutrisiran, were studied for their effects on transthyretin amyloid cardiomyopathy (ATTR-CA) by analyzing SPECT/CT imaging outcomes.
- Eight patients with hereditary ATTR-CA were monitored, with one group starting RNAi treatment alongside their first imaging, and another group being on treatment prior to imaging.
- Findings revealed a significant decrease in a volumetric heart/lung ratio in patients receiving RNAi therapies, indicating potential benefits in managing ATTR-CA symptoms.
Assessing the effectiveness and safety of Patisiran and Vutrisiran in ATTRv amyloidosis with polyneuropathy: a systematic review.
Front Neurol
September 2024
School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran.
Article Synopsis
- * A thorough literature review assessed 858 studies, resulting in the selection of 10 studies involving 756 patients, revealing both therapies improved neuropathy, quality of life, and cardiac function, with manageable side effects.
- * The systematic review supports the use of Patisiran and Vutrisiran for treating ATTRv, showing significant benefits and good safety profiles, but it calls for more extensive future studies due to potential biases and limited follow-up time in some research. *
Efficacy and safety of patisiran for ATTRv-PN: a systematic review and meta-analysis.
Ther Adv Neurol Disord
September 2024
Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, 12# Wulumuqi Zhong Road, Shanghai 200040, China.
Background: Hereditary transthyretin amyloidosis (ATTRv; v for variant) with polyneuropathy is a rare, progressive, and fatal autosomal dominant disorder. Therapies such as liver transplantation and TTR stabilizations have limitations. Patisiran is a small interfering RNA (siRNA), offering potential as a genetic-level therapy for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
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