4.8 Article

A circulating, disease-specific, mechanism-linked biomarker for ATTR polyneuropathy diagnosis and response to therapy prediction

Related references

Note: Only part of the references are listed.
Article Clinical Neurology

Neurofilament Light Chain as a Biomarker of Hereditary Transthyretin-Mediated Amyloidosis

Simina Ticau et al.

Summary: This study identified changes in proteome associated with onset and progression of hATTR amyloidosis, with findings suggesting that NfL may serve as a biomarker of nerve damage and polyneuropathy in ATTRv amyloidosis, enabling earlier diagnosis and disease progression monitoring. Results showed patisiran treatment trending the proteome of patients towards healthy controls and significantly correlating with reduced NfL levels and neuropathy impairment score improvement.

NEUROLOGY (2021)

Article Cell Biology

Does protein aggregation drive postmitotic tissue degeneration?

Jeffery W. Kelly

Summary: Pharmacological evidence from clinical trials suggests that protein aggregation plays a key role in tissue degeneration in systemic amyloid diseases, with ongoing investigations focusing on the protein aggregate structures driving tissue pathology and the similarities in etiology between these diseases and Alzheimer's disease.

SCIENCE TRANSLATIONAL MEDICINE (2021)

Article Multidisciplinary Sciences

Cryo-EM structure of a transthyretin-derived amyloid fibril from a patient with hereditary ATTR amyloidosis

Matthias Schmidt et al.

NATURE COMMUNICATIONS (2019)

Article Medicine, Research & Experimental

Predictive model of response to tafamidis in hereditary ATTR polyneuropathy

Ceclia Monteiro et al.

JCI INSIGHT (2019)

Article Clinical Neurology

Age-dependent cognitive dysfunction in untreated hereditary transthyretin amyloidosis

Ana Martins da Silva et al.

JOURNAL OF NEUROLOGY (2018)

Article Clinical Neurology

Estimating the global prevalence of transthyretin familial amyloid polyneuropathy

Hartmut H. Schmidt et al.

MUSCLE & NERVE (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)

Editorial Material Medicine, General & Internal

Oligonucleotide Drugs for Transthyretin Amyloidosis

Joel N. Buxbaum

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Medicine, General & Internal

Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy

Mathew S. Maurer et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Cell Biology

Peptide probes detect misfolded transthyretin oligomers in plasma of hereditary amyloidosis patients

Joseph D. Schonhoft et al.

SCIENCE TRANSLATIONAL MEDICINE (2017)

Article Biochemistry & Molecular Biology

Cognitive impairment in liver transplanted patients with transthyretin-related hereditary amyloid polyneuropathy

Vanda Freitas Castro et al.

AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2017)

Article Clinical Neurology

Cutaneous Nerve Biomarkers in Transthyretin Familial Amyloid Polyneuropathy

Gigi J. Ebenezer et al.

ANNALS OF NEUROLOGY (2017)

Article Biochemistry & Molecular Biology

Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy

Marcia Waddington Cruz et al.

AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2016)

Review Gastroenterology & Hepatology

Liver Transplantation in Transthyretin Amyloidosis: Issues and Challenges

Andreia Carvalho et al.

LIVER TRANSPLANTATION (2015)

Review Biotechnology & Applied Microbiology

Targeting protein aggregation for the treatment of degenerative diseases

Yvonne S. Eisele et al.

NATURE REVIEWS DRUG DISCOVERY (2015)

Review Nutrition & Dietetics

Plasma Transthyretin as a Biomarker of Lean Body Mass and Catabolic States

Yves Ingenbleek et al.

ADVANCES IN NUTRITION (2015)

Article Clinical Neurology

CNS involvement in V30M transthyretin amyloidosis: clinical, neuropathological and biochemical findings

Luis F. Maia et al.

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2015)

Article Medicine, General & Internal

Repurposing Diflunisal for Familial Amyloid Polyneuropathy A Randomized Clinical Trial

John L. Berk et al.

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2013)

Article Medicine, General & Internal

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

Teresa Coelho et al.

NEW ENGLAND JOURNAL OF MEDICINE (2013)

Article Multidisciplinary Sciences

Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade

Christine E. Bulawa et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)

Article Multidisciplinary Sciences

Atomic View of a Toxic Amyloid Small Oligomer

Arthur Laganowsky et al.

SCIENCE (2012)

Article Gastroenterology & Hepatology

Liver Transplantation for Familial Amyloidotic Polyneuropathy: Impact on Swedish Patients' Survival

Sadahisa Okamoto et al.

LIVER TRANSPLANTATION (2009)

Article Biochemistry & Molecular Biology

Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis

Yoshiki Sekijima et al.

AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2006)

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)

Article Multidisciplinary Sciences

Sequence-dependent denaturation energetics:: A major determinant in amyloid disease diversity

P Hammarström et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)

Article Multidisciplinary Sciences

Trans-suppression of misfolding in an amyloid disease

P Hammarström et al.

SCIENCE (2001)

Article Biochemistry & Molecular Biology

An engineered transthyretin monomer that is nonamyloidogenic, unless it is partially denatured

X Jiang et al.

BIOCHEMISTRY (2001)