Early biochemical effects of velmanase alfa in a 7‐month‐old infant with alpha‐mannosidosis by Lucia Santoro, Lucia Zampini, Lucia Padella, Chiara Monachesi, Stefania Zampieri, Andrea Dardis, Rosanna Cordiali, Tiziana Galeazzi, Carlo Catassi

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Cardiac involvement in MPS patients: incidence and response to therapy in an Italian multicentre study by Simona Sestito, Giada Rinninella, Angelica Rampazzo, Francesca D’Avanzo, Lucia Zampini, Lucia Santoro, Orazio Gabrielli, Agata Fiumara, Rita Barone, Nicola Volpi, Maurizio Scarpa, Rosella Tomanin, Daniela Concolino

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Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience by Rita Fischetto, Valentina Palladino, Maria M. Mancardi, Thea Giacomini, Stefano Palladino, Alberto Gaeta, Maja Di Rocco, Lucia Zampini, Giuseppe Lassandro, Vito Favia, Maria E. Tripaldi, Pietro Strisciuglio, Alfonso Romano, Mariasavina Severino, Amelia Morrone, Paola Giordano

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