TTR kinetic stabilizers and TTR gene silencing: a new era in therapy for familial amyloidotic polyneuropathies

Sixty years of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Europe

(2016) Yesim Parman et al.   CURRENT OPINION IN NEUROLOGY

Hereditary ATTR amyloidosis: a single-institution experience with 266 patients

(2015) Paul L. Swiecicki et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Safety and efficacy of long-term diflunisal administration in hereditary transthyretin (ATTR) amyloidosis

(2015) Yoshiki Sekijima et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Evolving landscape in the management of transthyretin amyloidosis

(2015) Philip N. Hawkins et al.   ANNALS OF MEDICINE

Genotype-phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France

(2015) Louise-Laure Mariani et al.   ANNALS OF NEUROLOGY

The Val30Met familial amyloid polyneuropathy specific Rasch-built overall disability scale (FAP-RODS©)

(2015) Mariëlle H. J. Pruppers et al.   JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM

Rapid progression of familial amyloidotic polyneuropathy

(2015) David Adams et al.   NEUROLOGY

Therapeutic Clearance of Amyloid by Antibodies to Serum Amyloid P Component

(2015) Duncan B. Richards et al.   NEW ENGLAND JOURNAL OF MEDICINE

Characterization of conformation-specific, human-derived monoclonal antibodies against TTR aggregates with potential for diagnostic and therapeutic use

(2015) Aubin Michalon et al.   Orphanet Journal of Rare Diseases

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

(2015) Ole B Suhr et al.   Orphanet Journal of Rare Diseases

Liver Transplantation for Hereditary Transthyretin Amyloidosis

(2015) Bo-Göran Ericzon et al.   TRANSPLANTATION

Repurposing Diflunisal for Familial Amyloid Polyneuropathy

(2015) John L. Berk et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Impact of liver transplantation on the natural history of oculopathy in Portuguese patients with transthyretin (V30M) amyloidosis

(2014) João Melo Beirão et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

In vivo detection of nerve injury in familial amyloid polyneuropathy by magnetic resonance neurography

(2014) Jennifer Kollmer et al.   BRAIN

Frequencies and geographic distributions of genetic mutations in transthyretin- and non-transthyretin-related familial amyloidosis

(2014) D.B. Zhen et al.   CLINICAL GENETICS

FAP Neuropathy and Emerging Treatments

(2014) David Adams et al.   Current Neurology and Neuroscience Reports

Online Registry for Mutations in Hereditary Amyloidosis Including Nomenclature Recommendations

(2014) Dorota M. Rowczenio et al.   HUMAN MUTATION

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

(2014) L. F. Maia et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Retrospective study of a TTR FAP cohort to modify NIS+7 for therapeutic trials

(2014) N. Suanprasert et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Transthyretin Induces Insulin-like Growth Factor I Nuclear Translocation Regulating Its Levels in the Hippocampus

(2014) Marta Vieira et al.   MOLECULAR NEUROBIOLOGY

An amyotrophic lateral sclerosis-like syndrome revealing an amyloid polyneuropathy associated with a novel transthyretin mutation

(2013) Pierre Lozeron et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Reduced Myocardial 123-Iodine Metaiodobenzylguanidine Uptake

(2013) Maria C. Azevedo Coutinho et al.   Circulation-Cardiovascular Imaging

Effect on disability and safety of Tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy

(2013) P. Lozeron et al.   EUROPEAN JOURNAL OF NEUROLOGY

Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy

(2013) Teresa Coelho et al.   JOURNAL OF NEUROLOGY

Diagnostic hallmarks and pitfalls in late-onset progressive transthyretin-related amyloid-neuropathy

(2013) Maike F. Dohrn et al.   JOURNAL OF NEUROLOGY

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effects of Tafamidis on Transthyretin Stabilization and Clinical Outcomes in Patients with Non-Val30Met Transthyretin Amyloidosis

(2013) Giampaolo Merlini et al.   Journal of Cardiovascular Translational Research

Regional difference and similarity of familial amyloidosis with polyneuropathy in France

(2012) David Adams et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy

(2012) Elizabeth J. Ackermann et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Amyloid neuropathies

(2012) David Adams et al.   CURRENT OPINION IN NEUROLOGY

The Transthyretin Amyloidoses: From Delineating the Molecular Mechanism of Aggregation Linked to Pathology to a Regulatory-Agency-Approved Drug

(2012) Steven M. Johnson et al.   JOURNAL OF MOLECULAR BIOLOGY

Natural history of transthyretin Val30Met familial amyloid polyneuropathy: analysis of late-onset cases from non-endemic areas

(2012) Haruki Koike et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

TTR-related amyloid neuropathy: clinical, electrophysiological and pathological findings in 15 unrelated patients

(2012) Marco Luigetti et al.   NEUROLOGICAL SCIENCES

Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial

(2012) T. Coelho et al.   NEUROLOGY

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

(2012) C. E. Bulawa et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Cardiac phenotype and clinical outcome of familial amyloid polyneuropathy associated with transthyretin alanine 60 variant

(2011) P. T. Sattianayagam et al.   EUROPEAN HEART JOURNAL

Clinical and histopathological features of familial amyloidotic polyneuropathy with transthyretin Val30Ala in a Chinese family

(2011) Jing-Yao Liu et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Antibodies to human serum amyloid P component eliminate visceral amyloid deposits

(2010) Karl Bodin et al.   NATURE

Clinical presentations and skin denervation in amyloid neuropathy due to transthyretin Ala97Ser

(2010) N. C.- C. Yang et al.   NEUROLOGY

Progression of transthyretin amyloid neuropathy after liver transplantation

(2010) J. J. Liepnieks et al.   NEUROLOGY

Transthyretin-related familial amyloidotic polyneuropathy—Progress in Kumamoto, Japan (1967–2010)—

(2010) Shukuro ARAKI et al.   PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES

Epidemiology of familial amyloid polyneuropathy in Japan: Identification of a novel endemic focus

(2008) Yuko Kato-Motozaki et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES