Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
David Adams,Alejandra González-Duarte,William O'Riordan,Chih-Chao Yang,Mitsuharu Ueda,Arnt V. Kristen,Ivailo Tournev,Hartmut Schmidt,Teresa Coelho,John L. Berk,Kon Ping Lin,Giuseppe Vita,Shahram Attarian,Violaine Planté-Bordeneuve,Michelle M. Mezei,Josep M. Campistol,Juan Buades,Thomas H. Brannagan,Byoung Joon Kim,Jeeyoung Oh,Yesim Parman,Yoshiki Sekijima,Philip N. Hawkins,Scott D. Solomon,Michael Polydefkis,Peter J. Dyck,Pritesh Gandhi,Sunita Goyal,Jihong Chen,Andrew Strahs,Saraswathy V. Nochur,Marianne T. Sweetser,Pushkal Garg,Pushkal Garg,Akshay Vaishnaw,Akshay Vaishnaw,Jared Gollob,Ole B. Suhr +37 more
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TLDR
Patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis with polyneuropathy and showed an effect on gait speed and modified BMI.Abstract:
BACKGROUND: Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin.METHODS: In this phase 3 trial, we randomly assigned patients ...read more
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2022 AHA/ACC/HFSA Guideline for the Management of Heart Failure: A Report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines.
Paul A. Heidenreich,Biykem Bozkurt,David Aguilar,Larry A. Allen,Joni J. Byun,Monica Colvin,Anita Deswal,Mark H. Drazner,Shannon M. Dunlay,Linda R. Evers,James C. Fang,Savitri Fedson,Gregg C. Fonarow,Salim S. Hayek,Adrian F. Hernandez,Prateeti Khazanie,Michelle M. Kittleson,Christopher S. Lee,Mark S. Link,Carmelo A. Milano,Lorraine C. Nnacheta,Alexander T. Sandhu,Lynne W. Stevenson,Orly Vardeny,Amanda R. Vest,Clyde W. Yancy +25 more
TL;DR: The "2022 AHA/ACC/HFSA Guideline for the Management of Heart Failure" as discussed by the authors provides patient-centric recommendations for clinicians to prevent, diagnose, and manage patients with heart failure.
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Nanoparticles in the clinic: An update.
TL;DR: A 3 year update on the current clinical landscape of nanoparticle drug delivery systems and highlight newly approved nanomedicines, provide a status update on previous clinical trials, and highlight new technologies that have recently entered the clinic are provided.
Journal ArticleDOI
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
Merrill D. Benson,Márcia Waddington-Cruz,John L. Berk,Michael Polydefkis,Peter J. Dyck,Annabel K. Wang,Violaine Planté-Bordeneuve,Fabio Barroso,Giampaolo Merlini,Laura Obici,Morton A. Scheinberg,Thomas H. Brannagan,William J. Litchy,Carol J. Whelan,Brian M. Drachman,David C. Adams,Stephen B. Heitner,Isabel Conceição,Hartmut H. Schmidt,Giuseppe Vita,Josep M. Campistol,Josep Gamez,Peter D. Gorevic,Edward Gane,Amil M. Shah,Scott D. Solomon,Brett P. Monia,Steven G. Hughes,Jesse Kwoh,Bradley W. McEvoy,Shiangtung W. Jung,Brenda F. Baker,Elizabeth J. Ackermann,Morie A. Gertz,Teresa Coelho +34 more
TL;DR: Inotersen improved the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis and improvements were independent of disease stage, mutation type, or the presence of cardiomyopathy.
Journal ArticleDOI
The current state and future directions of RNAi-based therapeutics.
TL;DR: This Review discusses key advances in the design and development of RNAi drugs leading up to this landmark achievement, the state of the current clinical pipeline and prospects for future advances, including novel RNAi pathway agents utilizing mechanisms beyond post-translational RNAi silencing.
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The Role of Non-coding RNAs in Oncology
TL;DR: For decades, research into cancer biology focused on the involvement of protein-coding genes, but an explosion of studies into ncRNA biology has shown that they represent a diverse and prevalent group of RNAs, including both oncogenic molecules and those that work in a tumor suppressive manner.
References
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Journal ArticleDOI
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
Merrill D. Benson,Márcia Waddington-Cruz,John L. Berk,Michael Polydefkis,Peter J. Dyck,Annabel K. Wang,Violaine Planté-Bordeneuve,Fabio Barroso,Giampaolo Merlini,Laura Obici,Morton A. Scheinberg,Thomas H. Brannagan,William J. Litchy,Carol J. Whelan,Brian M. Drachman,David C. Adams,Stephen B. Heitner,Isabel Conceição,Hartmut H. Schmidt,Giuseppe Vita,Josep M. Campistol,Josep Gamez,Peter D. Gorevic,Edward Gane,Amil M. Shah,Scott D. Solomon,Brett P. Monia,Steven G. Hughes,Jesse Kwoh,Bradley W. McEvoy,Shiangtung W. Jung,Brenda F. Baker,Elizabeth J. Ackermann,Morie A. Gertz,Teresa Coelho +34 more
TL;DR: Inotersen improved the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis and improvements were independent of disease stage, mutation type, or the presence of cardiomyopathy.
Journal ArticleDOI
Safety and efficacy of RNAi therapy for transthyretin amyloidosis
Teresa Coelho,David H. Adams,Ana Martins da Silva,Pierre Lozeron,Philip N. Hawkins,Timothy Mant,Javier Perez,Joseph Chiesa,Steve Warrington,Elizabeth Tranter,Malathy Munisamy,Rick Falzone,Jamie Harrop,Jeffrey Cehelsky,Brian Bettencourt,Mary Geissler,James Butler,Alfica Sehgal,Rachel Meyers,Qingmin Chen,Todd Borland,Renta Hutabarat,Valerie A. Clausen,Rene Alvarez,Kevin Fitzgerald,Christina Gamba-Vitalo,Saraswathy V. Nochur,Akshay Vaishnaw,Dinah W.Y. Sah,Jared Gollob,Ole B. Suhr +30 more
TL;DR: A therapeutic approach mediated by RNA interference (RNAi) could reduce the production of transthyretin in peripheral nerves and the heart as mentioned in this paper, which is caused by the deposition of hepatocyte-derived tranthymretin amyloid in peripheral nerve and heart.
Journal ArticleDOI
Repurposing Diflunisal for Familial Amyloid Polyneuropathy A Randomized Clinical Trial
John L. Berk,Ole B. Suhr,Laura Obici,Yoshiki Sekijima,Steven R. Zeldenrust,Taro Yamashita,Michael A. Heneghan,Peter D. Gorevic,William J. Litchy,Janice F. Wiesman,Erik Nordh,Manuel Corato,Alessandro Lozza,Andrea Cortese,Jessica Robinson-Papp,Theodore Colton,Denis Rybin,Alice Bisbee,Yukio Ando,Shu-ichi Ikeda,David C. Seldin,Giampaolo Merlini,Martha Skinner,Jeffery W. Kelly,Peter J. Dyck +24 more
TL;DR: Among patients with familial amyloid polyneuropathy, the use of diflunisal compared with placebo for 2 years reduced the rate of progression of neurological impairment and preserved quality of life.
Journal ArticleDOI
Guideline of transthyretin-related hereditary amyloidosis for clinicians
Yukio Ando,Teresa Coelho,John L. Berk,Márcia Waddington Cruz,Bo Göran Ericzon,Shu-ichi Ikeda,W. David Lewis,Laura Obici,Violaine Planté-Bordeneuve,Claudio Rapezzi,Gérard Said,Fabrizio Salvi +11 more
TL;DR: Providing guidance on making a definitive diagnosis, methods for disease staging and evaluation of disease progression, and discuss symptom mitigation and treatment strategies, including liver transplant and several pharmacotherapies that have shown promise in clinical trials are provided.
Journal ArticleDOI
Transthyretin (TTR) Cardiac Amyloidosis
Frederick L. Ruberg,John L. Berk +1 more
TL;DR: The systemic amyloidoses are a family of diseases induced by misfolded or misassembled proteins that can infiltrate the heart, resulting in progressive diastolic and systolic dysfunction, congestive heart failure, and death.
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