Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System.

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作者： Silvia,Pontesilli ^[1] ; Cristina,Baldoli ^[1] ; Pasquale Anthony Della,Rosa ^[1] ; Alessandro,Cattoni ^[2] ; Maria Ester,Bernardo ^[3] ; Pamela,Meregalli ^[2] ; Serena,Gasperini ^[2] ; Serena,Motta ^[2] ; Francesca,Fumagalli ^[4] ; Francesca,Tucci ^[4] ; Federica,Baciga ^[4] ; Giulia,Consiglieri ^[4] ; Francesco,Canonico ^[5] ; Paola,De Lorenzo ^[6] ; Luisa,Chiapparini ^[7] ; Bernhard,Gentner ^[3] ; Alessandro,Aiuti ^[3] ; Andrea,Biondi ^[2] ; Attilio,Rovelli ^[2] ; Rossella,Parini ^[8]

作者单位： Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy. ^[1] Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy. ^[2] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy; Vita-Salute San Raffaele University of Milano, Milano, Italy. ^[3] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy. ^[4] Department of Radiology, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy. ^[5] Centro Operativo di Ricerca Statistica, Fondazione Tettamanti, University of Milano-Bicocca, Monza, Italy. ^[6] Neuroradiology Unit, IRCCS Istituto Neurologico Carlo Besta, Milano, Italy. ^[7] Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy. Electronic address: rossella.parini@unimib.it. ^[8]

关键词 ERT and MPS HSCT MPSI MPSI-H haematopoietic stem cell transplantation magnetic resonance neuroradiology and MPS