Familial TTR amyloid polyneuropathy (FAP TTR): treatment
Evidence-based neurology checklist on familial ttr amyloid polyneuropathy (fap ttr): treatment: Liver transplantation: options Combined kidney-liver transplant Combined heart-liver transplant Domino liver transplant Tafamidis Diflunisal Inotersen Patisiran Vitrusiran Neuropathic treatments Other…
Liver transplantation: options
- Combined kidney-liver transplant
- Combined heart-liver transplant
- Domino liver transplant
Tafamidis
Diflunisal
Inotersen
Patisiran
Vitrusiran
Neuropathic treatments
Other symptomatic treatments
Pre-symptomatic management
Investigational treatments
References
- Ando Y, Coelho T, Berk JL, et al. Guideline for transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis 2013; 8:31.
- Coelho T, Maia LF, da Silva AM, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology 2012; 79:785-792.
- Cortese A, Vita G, Luigetti M, et al. Monitoring effectiveness and safety of Tafamidis in transthyretin amyloidosis in Italy: a longitudinal multicenter study in a non-endemic area. J Neurol 2016; 263:916-924.
- Conceição I, Miranda B, Castro J, de Carvalho M. Hereditary amyloidosis related to transthyretin V30M: disease progression in treated and untreated patients. Eur J Neurol 2018; 25:1320-e115.
- Planté-Bordeneuve V, Gorram F, Salhi H, et al. Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study. J Neurol 2017; 264:268-276.
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