[HTML][HTML] Hereditary Transthyretin Amyloidosis with Polyneuropathy: Monitoring and Management

V Vélez-Santamaría, V Nedkova-Hristova… - … Journal of General …, 2022 - ncbi.nlm.nih.gov
Hereditary Transthyretin Amyloidosis with Polyneuropathy: Monitoring and Management - PMC
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Current and future treatment approaches in transthyretin familial amyloid polyneuropathy

P Kerschen, V Planté-Bordeneuve - Current treatment options in neurology, 2016 - Springer
Opinion statement Treatment of transthyretin familial amyloid polyneuropathy (TTR FAP)
must be tailored to disease stage. Patients with early stage disease (ie, without major …

[HTML][HTML] Long-term treatment of hereditary transthyretin amyloidosis with patisiran: multicentre, real-world experience in Italy

L Gentile, A Mazzeo, C Briani, S Casagrande… - Neurological …, 2024 - Springer
Background Hereditary transthyretin (ATTRv, v for variant) amyloidosis with polyneuropathy
is a rare disease caused by mutations in the transthyretin gene. In ATTRv amyloidosis …

Canadian guidelines for hereditary transthyretin amyloidosis polyneuropathy management

M Alcantara, MM Mezei, SK Baker, A Breiner… - Canadian Journal of …, 2022 - cambridge.org
Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive disease caused by
mutations in the TTR gene leading to multisystem organ dysfunction. Pathogenic TTR …

Novel approaches to diagnosis and management of hereditary transthyretin amyloidosis

A Carroll, PJ Dyck, M de Carvalho… - Journal of Neurology …, 2022 - jnnp.bmj.com
Hereditary transthyretin amyloidosis (ATTRv) is a severe, adult-onset autosomal dominant
inherited systemic disease predominantly affecting the peripheral and autonomic nervous …

Diagnosis and treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy: current perspectives on improving patient care

M Luigetti, A Romano, A Di Paolantonio… - … and clinical risk …, 2020 - Taylor & Francis
Hereditary transthyretin amyloidosis (hATTR) with polyneuropathy (formerly known as
Familial Amyloid Polyneuropathy) is a rare disease due to mutations in the gene encoding …

Epidemiological and clinical characteristics of persons with transthyretin hereditary amyloid polyneuropathy: a global synthesis of 532 cases

MW Cruz, H Schmidt, MF Botteman, JA Carter… - Amyloid, 2017 - Taylor & Francis
Background Transthyretin hereditary (familial) amyloid polyneuropathy (TTR-FAP) was first
identified in Portugal in 1952 [Citation 1]. In 1984, the first report of a causative mutation …

[HTML][HTML] Chance or challenge, spoilt for choice? New recommendations on diagnostic and therapeutic considerations in hereditary transthyretin amyloidosis with …

MF Dohrn, M Auer-Grumbach, R Baron, F Birklein… - Journal of …, 2021 - Springer
Hereditary transthyretin amyloidosis is caused by pathogenic variants (ATTR v) in the TTR
gene. Alongside cardiac dysfunction, the disease typically manifests with a severely …

An evaluation of patisiran: a viable treatment option for transthyretin-related hereditary amyloidosis

P Milani, R Mussinelli, S Perlini… - Expert Opinion on …, 2019 - Taylor & Francis
Introduction: Hereditary transthyretin-mediated amyloidosis (ATTRv; v for variant) is a rare,
progressive, fatal multi-systemic disease, autosomal dominantly inherited with …

Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease

D Adams, H Koike, M Slama, T Coelho - Nature Reviews Neurology, 2019 - nature.com
Hereditary amyloidogenic transthyretin (ATTRv) amyloidosis with polyneuropathy (also
known as familial amyloid polyneuropathy) is a condition with adult onset caused by …