Your browser doesn't support javascript.
loading
Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience.
Fischetto, Rita; Palladino, Valentina; Mancardi, Maria M; Giacomini, Thea; Palladino, Stefano; Gaeta, Alberto; Di Rocco, Maja; Zampini, Lucia; Lassandro, Giuseppe; Favia, Vito; Tripaldi, Maria E; Strisciuglio, Pietro; Romano, Alfonso; Severino, Mariasavina; Morrone, Amelia; Giordano, Paola.
Afiliação
  • Fischetto R; Clinical Genetics Unit, Department of Pediatric Medicine, Giovanni XXIII Children's Hospital, Bari, Italy.
  • Palladino V; Department of Biomedical Science and Human Oncology, Pediatric Unit, University of Bari "Aldo Moro", Bari, Italy.
  • Mancardi MM; Unit of Child Neuropsychiatry, Clinical and Surgical Neurosciences Department, IRCCS Institute Giannina Gaslini, Genoa, Italy.
  • Giacomini T; Unit of Child Neuropsychiatry, Clinical and Surgical Neurosciences Department, IRCCS Institute Giannina Gaslini, Genoa, Italy.
  • Palladino S; Radiology Unit, Pediatric Hospital Giovanni XXIII, Bari, Italy.
  • Gaeta A; Radiology Unit, Pediatric Hospital Giovanni XXIII, Bari, Italy.
  • Di Rocco M; Unit of Rare Diseases, IRCCS Institute Giannina Gaslini, Genoa, Italy.
  • Zampini L; Department of Clinical Sciences, Division of Pediatrics, Polytechnic University of Marche, OspedaliRiuniti, Presidio Salesi, Ancona, Italy.
  • Lassandro G; Department of Biomedical Science and Human Oncology, Pediatric Unit, University of Bari "Aldo Moro", Bari, Italy.
  • Favia V; Clinical Genetics Unit, Department of Pediatric Medicine, Giovanni XXIII Children's Hospital, Bari, Italy.
  • Tripaldi ME; Department of Biomedical Science and Human Oncology, Pediatric Unit, University of Bari "Aldo Moro", Bari, Italy.
  • Strisciuglio P; Department of Medical Translational Sciences Section of Pediatrics, University Federico II Naples, Napoli, Italy.
  • Romano A; Department of Medical Translational Sciences Section of Pediatrics, University Federico II Naples, Napoli, Italy.
  • Severino M; Neuroradiology Unit, IRCCS Institute Giannina Gaslini, Genoa, Italy.
  • Morrone A; Paediatric Neurology Unit and Laboratories, Neuroscience Department, Meyer Children's Hospital, Florence, Italy.
  • Giordano P; Department of Biomedical Science and Human Oncology, Pediatric Unit, University of Bari "Aldo Moro", Bari, Italy.
Mol Genet Genomic Med ; 8(10): e1371, 2020 10.
Article em En | MEDLINE | ID: mdl-32779865

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Gangliosidose GM1 / 1-Desoxinojirimicina / Inibidores de Glicosídeo Hidrolases Tipo de estudo: Clinical_trials Limite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Mol Genet Genomic Med Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Itália

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Gangliosidose GM1 / 1-Desoxinojirimicina / Inibidores de Glicosídeo Hidrolases Tipo de estudo: Clinical_trials Limite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Mol Genet Genomic Med Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Itália