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Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System.
Pontesilli, Silvia; Baldoli, Cristina; Rosa, Pasquale Anthony Della; Cattoni, Alessandro; Bernardo, Maria Ester; Meregalli, Pamela; Gasperini, Serena; Motta, Serena; Fumagalli, Francesca; Tucci, Francesca; Baciga, Federica; Consiglieri, Giulia; Canonico, Francesco; De Lorenzo, Paola; Chiapparini, Luisa; Gentner, Bernhard; Aiuti, Alessandro; Biondi, Andrea; Rovelli, Attilio; Parini, Rossella.
Afiliação
  • Pontesilli S; Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.
  • Baldoli C; Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.
  • Rosa PAD; Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.
  • Cattoni A; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.
  • Bernardo ME; 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.
  • Meregalli P; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.
  • Gasperini S; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.
  • Motta S; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.
  • Fumagalli F; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.
  • Tucci F; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.
  • Baciga F; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.
  • Consiglieri G; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milano, Italy.
  • Canonico F; Department of Radiology, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.
  • De Lorenzo P; Centro Operativo di Ricerca Statistica, Fondazione Tettamanti, University of Milano-Bicocca, Monza, Italy.
  • Chiapparini L; Neuroradiology Unit, IRCCS Istituto Neurologico Carlo Besta, Milano, Italy.
  • Gentner B; 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.
  • Aiuti A; 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.
  • Biondi A; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.
  • Rovelli A; Pediatric Department, Fondazione MBBM, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy.
  • Parini R; 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.
J Pediatr ; 240: 297-301.e5, 2022 01.
Article em En | MEDLINE | ID: mdl-34547335
We developed a brain and spine magnetic resonance scoring system based on a magnetic resonance assessment of 9 patients with mucopolysaccharidosis type I-Hurler who underwent hematopoietic stem-cell transplantation. The score is reliable and correlates with long-term clinical and cognitive outcome in patients with mucopolysaccharidosis type I-Hurler.
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Medula Espinal / Encéfalo / Mucopolissacaridose I Tipo de estudo: Observational_studies / Prognostic_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Medula Espinal / Encéfalo / Mucopolissacaridose I Tipo de estudo: Observational_studies / Prognostic_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Ano de publicação: 2022 Tipo de documento: Article