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Long-term treatment of hereditary transthyretin amyloidosis with patisiran: multicentre, real-world experience in Italy
Hereditary transthyretin (ATTRv, v for variant) amyloidosis with polyneuropathy is a rare disease caused by mutations in the transthyretin gene. In ATTRv amyloidosis, multisystem extracellular deposits of amyl...
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Hereditary Transthyretin Amyloidosis
Hereditary transthyretin amyloidosis (ATTRv) is a severe, adult-onset, autosomal dominant systemic disease caused by mutations in the TTR gene. Mutations destabilize the native tetrameric structure of the transpo...
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Article
Open AccessMuscle quantitative MRI as a novel biomarker in hereditary transthyretin amyloidosis with polyneuropathy: a cross-sectional study
The development of reproducible and sensitive outcome measures has been challenging in hereditary transthyretin (ATTRv) amyloidosis. Recently, quantification of intramuscular fat by magnetic resonance imaging ...
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Article
Current and Emerging Therapies for Hereditary Transthyretin Amyloidosis: Strides Towards a Brighter Future
The past few years have witnessed an unprecedented acceleration in the clinical development of novel therapeutic options for hereditary transthyretin amyloidosis. Recently approved agents and drugs currently u...
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Article
New and Evolving Concepts Regarding the Prognosis and Treatment of Cardiac Amyloidosis
Systemic amyloidoses are rare and proteiform diseases, caused by extracellular accumulation of insoluble misfolded fibrillar proteins. Prognosis is dictated by cardiac involvement, which is especially frequent...
