4.7 Article

Pathophysiology and Therapeutic Approaches to Cardiac Amyloidosis

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Biochemistry & Molecular Biology

Blinded potency comparison of transthyretin kinetic stabilisers by subunit exchange in human plasma

Luke T. Nelson et al.

Summary: The research indicates that the drug candidates AG10, tolcapone, diflunisal, and tafamidis exhibit similar kinetic stabilization at specific concentrations, with differences in safety, absorption and metabolism, pharmacokinetics, and tissue distribution becoming important factors for clinical use decisions regarding kinetic stabilizers.

AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2021)

Article Cardiac & Cardiovascular Systems

Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data

Nicholas J. Viney et al.

Summary: AKCEA-TTR-L-Rx, a ligand-conjugated antisense drug, showed significantly increased potency compared to unconjugated inotersen in human hepatocyte cell culture and transgenic hTTR mice expressing a mutated human genomic TTR sequence. In a phase 1 study with healthy volunteers, AKCEA-TTR-L-Rx demonstrated a reduction in TTR levels and a favorable safety profile, supporting its further development for the treatment of ATTR polyneuropathy and cardiomyopathy.

ESC HEART FAILURE (2021)

Article Cardiac & Cardiovascular Systems

Cost-Effectiveness of Tafamidis Therapy for Transthyretin Amyloid Cardiomyopathy

Dhruv S. Kazi et al.

CIRCULATION (2020)

Review Pharmacology & Pharmacy

Targeting the ubiquitin-proteasome pathway to overcome anti-cancer drug resistance

Silpa Narayanan et al.

DRUG RESISTANCE UPDATES (2020)

Article Cardiac & Cardiovascular Systems

Stabilization of Cardiac Function With Diflunisal in Transthyretin (ATTR) Cardiac Amyloidosis

Graham Lohrmann et al.

JOURNAL OF CARDIAC FAILURE (2020)

Editorial Material Cardiac & Cardiovascular Systems

Tafamidis-A Pricey Therapy for a Not-So-Rare Condition

Jerry H. Gurwitz et al.

JAMA CARDIOLOGY (2020)

Article Cardiac & Cardiovascular Systems

Outcomes in Patients With Cardiac Amyloidosis Undergoing Heart Transplantation

Christopher D. Barrett et al.

JACC-HEART FAILURE (2020)

Article Medicine, General & Internal

Adjuvant doxycycline to enhance anti-amyloid effects: Results from the dual phase 2 trial

Anita D'Souza et al.

ECLINICALMEDICINE (2020)

Article Pharmacology & Pharmacy

The Bioequivalence of Tafamidis 61-mg Free Acid Capsules and Tafamidis Meglumine 4 x 20-mg Capsules in Healthy Volunteers

Peter A. Lockwood et al.

CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT (2020)

Review Hematology

Glucocorticoids in multiple myeloma: past, present, and future

Nicholas Burwick et al.

ANNALS OF HEMATOLOGY (2019)

Article Biochemistry & Molecular Biology

A pair of peptides inhibits seeding of the hormone transporter transthyretin into amyloid fibrils

Lorena Saelices et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2019)

Article Cardiac & Cardiovascular Systems

Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy

Daniel P. Judge et al.

JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY (2019)

Review Cell Biology

The proteostasis network and its decline in ageing

Mark S. Hipp et al.

NATURE REVIEWS MOLECULAR CELL BIOLOGY (2019)

Review Cardiac & Cardiovascular Systems

The pathology and changing epidemiology of dialysis-related cardiac beta-2 microglobulin amyloidosis

Allison D. Morris et al.

CARDIOVASCULAR PATHOLOGY (2019)

Article Multidisciplinary Sciences

Stabilization of amyloidogenic immunoglobulin light chains by small molecules

Gareth J. Morgan et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2019)

Article Biochemistry & Molecular Biology

Mechanistic Insights into the Early Events in the Aggregation of Immunoglobulin Light Chains

Pinaki Misra et al.

BIOCHEMISTRY (2019)

Review Cardiac & Cardiovascular Systems

Transthyretin Amyloid Cardiomyopathy JACC State-of-the-Art Review

Frederick L. Ruberg et al.

JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY (2019)

Article Cardiac & Cardiovascular Systems

Clinical Experience With the Use of Doxycycline and Ursodeoxycholic Acid for the Treatment of Transthyretin Cardiac Amyloidosis

Erin Karlstedt et al.

JOURNAL OF CARDIAC FAILURE (2019)

Meeting Abstract Biochemistry & Molecular Biology

One year follow up analysis of the phase 1a/b study of chimeric fibril-reactive monoclonal antibody 11-1F4 in patients with AL amyloidosis

Camille V. Edwards et al.

AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2019)

Review Cell Biology

RNA-Targeted Therapeutics

Stanley T. Crooke et al.

CELL METABOLISM (2018)

Article Cardiac & Cardiovascular Systems

Improved outcomes after heart transplantation for cardiac amyloidosis in the modern era

Arnt V. Kristen et al.

JOURNAL OF HEART AND LUNG TRANSPLANTATION (2018)

Article Medicine, General & Internal

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

D. Adams et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Medicine, General & Internal

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

M. D. Benson et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Multidisciplinary Sciences

Amyloid seeding of transthyretin by ex vivo cardiac fibrils and its inhibition

Lorena Saelices et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2018)

Article Medicine, General & Internal

Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy

Mathew S. Maurer et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Review Hematology

Venetoclax for the treatment of multiple myeloma

Iuliana Vaxman et al.

EXPERT REVIEW OF HEMATOLOGY (2018)

Article Biotechnology & Applied Microbiology

Cellular uptake and trafficking of antisense oligonucleotides

Stanley T. Crooke et al.

NATURE BIOTECHNOLOGY (2017)

Article Education, Scientific Disciplines

AL amyloidosis: from molecular mechanisms to targeted therapies

Giampaolo Merlini

HEMATOLOGY-AMERICAN SOCIETY OF HEMATOLOGY EDUCATION PROGRAM (2017)

Article Cardiac & Cardiovascular Systems

Addressing Common Questions Encountered in the Diagnosis and Management of Cardiac Amyloidosis

Mathew S. Maurer et al.

CIRCULATION (2017)

Article Chemistry, Multidisciplinary

Structure of Monomeric Transthyretin Carrying the Clinically Important T119M Mutation

Jin Hae Kim et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2016)

Review Immunology

Monoclonal antibodies targeting CD38 in hematological malignancies and beyond

Niels W. C. J. van de Donk et al.

IMMUNOLOGICAL REVIEWS (2016)

Review Multidisciplinary Sciences

The activities of amyloids from a structural perspective

Roland Riek et al.

NATURE (2016)

Article Multidisciplinary Sciences

Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity

Ricardo Sant'Anna et al.

NATURE COMMUNICATIONS (2016)

Article Biochemistry & Molecular Biology

Uncovering the Mechanism of Aggregation of Human Transthyretin

Lorena Saelices et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2015)

Article Medicine, General & Internal

Therapeutic Clearance of Amyloid by Antibodies to Serum Amyloid P Component

Duncan B. Richards et al.

NEW ENGLAND JOURNAL OF MEDICINE (2015)

Article Medicine, General & Internal

Targeting CD38 with Daratumumab Monotherapy in Multiple Myeloma

H. M. Lokhorst et al.

NEW ENGLAND JOURNAL OF MEDICINE (2015)

Article Genetics & Heredity

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Ole B. Suhr et al.

ORPHANET JOURNAL OF RARE DISEASES (2015)

Article Biochemistry & Molecular Biology

Age-Related Oxidative Modifications of Transthyretin Modulate Its Amyloidogenicity

Lei Zhao et al.

BIOCHEMISTRY (2013)

Article Multidisciplinary Sciences

AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin

Sravan C. Penchala et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2013)

Review Immunology

Amyloidosis in autoinflammatory syndromes

Laura Obici et al.

AUTOIMMUNITY REVIEWS (2012)

Review Biochemistry & Molecular Biology

The Amyloid State of Proteins in Human Diseases

David Eisenberg et al.

Article Multidisciplinary Sciences

Antibodies to human serum amyloid P component eliminate visceral amyloid deposits

Karl Bodin et al.

NATURE (2010)

Article Multidisciplinary Sciences

Amyloidogenic light chains induce cardiomyocyte contractile dysfunction and apoptosis via a non-canonical p38α MAPK pathway

Jianru Shi et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2010)

Review Biochemistry & Molecular Biology

Transthyretin: the servant of many masters

Joel N. Buxbaum et al.

CELLULAR AND MOLECULAR LIFE SCIENCES (2009)

Review Oncology

Melphalan and its role in the management of patients with multiple myeloma

Patrizia Falco et al.

EXPERT REVIEW OF ANTICANCER THERAPY (2007)

Article Biochemistry & Molecular Biology

The biological and chemical basis for tissue-selective amyloid disease

Y Sekijima et al.

Article Multidisciplinary Sciences

Tissue damage in the amyloidoses: Transthyretin monomers and nonnative oligomers are the major cytotoxic species in tissue culture

N Reixach et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2004)

Review Multidisciplinary Sciences

Protein folding and misfolding

CM Dobson

NATURE (2003)

Review Medicine, General & Internal

Molecular mechanisms of amyloidosis

G Merlini et al.

NEW ENGLAND JOURNAL OF MEDICINE (2003)

Article Multidisciplinary Sciences

Prevention of transthyretin amyloid disease by changing protein misfolding energetics

P Hammarström et al.

SCIENCE (2003)

Review Biochemistry & Molecular Biology

Review: Immunoglobulin light chain amyloidosis - The archetype of structural and pathogenic variability

V Bellotti et al.

JOURNAL OF STRUCTURAL BIOLOGY (2000)

Review Biochemistry & Molecular Biology

Immunoglobulin light chains, glycosaminoglycans, and amyloid

FJ Stevens et al.

CELLULAR AND MOLECULAR LIFE SCIENCES (2000)