The patient journey toward a diagnosis of hereditary transthyretin (ATTRv) amyloidosis

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[Familial approach in hereditary transthyretin cardiac amyloidosis].

Cardiac amyloidosis is a disease of complex diagnosis and treatment. Some subtypes of cardiac amyloidosis are inherited. Among these, the most common variant is caused by mutations in the transthyretin gene. Correct identification of amyloidosis produced by a genetic defect is of great importance because it modifies the diagnostic and therapeutic approach in patients and their families. We desc...

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Hereditary Transthyretin Amyloidosis in Eight Chinese Families

BACKGROUND Mutations of transthyretin (TTR) cause the most common type of autosomal-dominant hereditary systemic amyloidosis, which occurs worldwide. To date, more and more mutations in the TTR gene have been reported. Some variations in the clinical presentation are often observed in patients with the same mutation or the patients in the same family. The purpose of this study was to find out t...

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Successful Diflunisal Desensitization in a Transthyretin Amyloidosis Patient with NSAID Allergy: A Case Report

Introduction: Amyloid diseases have been known to be hereditary, including transthyretin (TTR) amyloidosis where subunit protein mutations may occur in genes for TTR leading to the deposition of fibrils (low molecular weight subunits (5 to 25 kD) of proteins) in extracellular tissues. By reducing the formation of TTR amyloid, diflunisal, a nonsteroidal anti-inflammatory drug (NSAID), has shown ...

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Diagnosis, Prognosis, and Therapy of Transthyretin Amyloidosis.

Transthyretin amyloidosis is a fatal disorder that is characterized primarily by progressive neuropathy and cardiomyopathy. It occurs in both a mutant form (with autosomal dominant inheritance) and a wild-type form (with predominant cardiac involvement). This article guides clinicians as to when the disease should be suspected, describes the appropriate diagnostic evaluation for those with know...

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ژورنال

عنوان ژورنال: Orphanet Journal of Rare Diseases

سال: 2021

ISSN: 1750-1172

DOI: 10.1186/s13023-020-01623-1