Patisiran in ATTRv amyloidosis with polyneuropathy: “PatisiranItaly” multicenter observational study - Di Stefano, Vincenzo; Guaraldi, Pietro; Romano, Angela; Antonini, Giovanni; Barilaro, Alessandro; Briani, Chiara; Burattini, Marco; Cani, Ilaria; Carlini, Giulia; Ceccanti, Marco; Cianci, Vittoria; Cortelli, Pietro; et al. (Journal of neurology, 2025)
Keywords: Adult; Aged; Amyloid Neuropathies, Familial - complications; Amyloid Neuropathies, Familial - drug therapy; Amyloid Neuropathies, Familial - genetics; Amyloidosis; Autonomic nervous system; Diabetes mellitus; Diabetic neuropathy; Double-stranded RNA; Female; Fibrils; Genotypes; Hepatocytes; Humans; Italy; Male; Medicine; Medicine & Public Health; Middle Aged; mRNA; Nanoparticles; Nerves; Neurology; Neuroradiology; Neurosciences; Observational studies; Original Communication; Polyneuropathies - drug therapy; Polyneuropathy; Prealbumin - genetics; Quality of Life; RNA, Small Interfering - therapeutic use; Sensorimotor system; Transthyretin; Treatment Outcome; Italy; Hereditary transthyretin amyloidosis; Patisiran; RNA interference; Multicenter study; ATTRv-PN; Real-world data