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18-year follow-up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I.
Pjetraj, Dorina; Santoro, Lucia; Sgattoni, Claudia; Padella, Lucia; Zampini, Lucia; Monachesi, Chiara; Gabrielli, Orazio; Catassi, Carlo.
  • Pjetraj D; Pediatric Division, Department of Clinical Sciences, Polytechnic University of Marche, Ospedali Riuniti, Presidio Salesi, Ancona, Italy.
  • Santoro L; Pediatric Division, Department of Clinical Sciences, Polytechnic University of Marche, Ospedali Riuniti, Presidio Salesi, Ancona, Italy.
  • Sgattoni C; Medical Genetics and Rare Disease Coordination, Institute of Maternal-Infantile Sciences, Ospedali Riuniti, Presidio Torrette, Ancona, Italy.
  • Padella L; Pediatric Division, Department of Clinical Sciences, Polytechnic University of Marche, Ospedali Riuniti, Presidio Salesi, Ancona, Italy.
  • Zampini L; Pediatric Division, Department of Clinical Sciences, Polytechnic University of Marche, Ospedali Riuniti, Presidio Salesi, Ancona, Italy.
  • Monachesi C; Pediatric Division, Department of Clinical Sciences, Polytechnic University of Marche, Ospedali Riuniti, Presidio Salesi, Ancona, Italy.
  • Gabrielli O; Pediatric Division, Department of Clinical Sciences, Polytechnic University of Marche, Ospedali Riuniti, Presidio Salesi, Ancona, Italy.
  • Catassi C; Pediatric Division, Department of Clinical Sciences, Polytechnic University of Marche, Ospedali Riuniti, Presidio Salesi, Ancona, Italy.
Am J Med Genet A ; 191(2): 564-569, 2023 02.
Article en En | MEDLINE | ID: mdl-36333985
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Mucopolisacaridosis I / Terapia de Reemplazo Enzimático Tipo de estudio: Screening_studies Límite: Child, preschool / Humans / Infant Idioma: En Año: 2023 Tipo del documento: Article
Texto completo
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XML
PubMed Links
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Mucopolisacaridosis I / Terapia de Reemplazo Enzimático Tipo de estudio: Screening_studies Límite: Child, preschool / Humans / Infant Idioma: En Año: 2023 Tipo del documento: Article