Luca Gentile, Anna Mazzeo, Chiara Briani, Silvia Casagrande, Marcella De Luca, Gian Maria Fabrizi, Christian Gagliardi, Chiara Gemelli, Francesca Forcina, Marina Grandis, Valeria Guglielmino, Giacomo Iabichella, Luca Leonardi, Alessandro Lozza, Fiore Manganelli, Roberta Mussinelli, Filomena My, Giuseppe Occhipinti, Silvia Fenu, Massimo Russo, Angela Romano, Alessandro Salvalaggio, Matteo Tagliapietra, Stefano Tozza, Giovanni Palladini, Laura Obici, Marco Luigetti
BACKGROUND: 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 amyloid cause tissue and organ dysfunction. Patisiran is a small interfering RNA molecule drug that reduces circulating levels of mutant and wild-type TTR proteins. Prior to its regulatory approval, patisiran was available in Italy through a compassionate use programme (CUP)...
April 16, 2024: Neurological Sciences