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First experience of combined enzyme replacement therapy and hematopoietic stem cell transplantation in alpha-mannosidosis.
Santoro, Lucia; Monachesi, Chiara; Zampini, Lucia; Padella, Lucia; Galeazzi, Tiziana; Santori, Elena; Cordiali, Rosanna; Dardis, Andrea; Catassi, Carlo; Boccieri, Emilia; Galaverna, Federica; Locatelli, Franco.
Affiliation
  • Santoro L; Division of Pediatrics, Department of Clinical Sciences, Azienda Ospedaliero Universitaria delle Marche, Presidio Salesi, Ancona, Italy.
  • Monachesi C; Division of Pediatrics, Department of Clinical Sciences, Azienda Ospedaliero Universitaria delle Marche, Presidio Salesi, Ancona, Italy.
  • Zampini L; Division of Pediatrics, Department of Clinical Sciences, Azienda Ospedaliero Universitaria delle Marche, Presidio Salesi, Ancona, Italy.
  • Padella L; Division of Pediatrics, Department of Clinical Sciences, Azienda Ospedaliero Universitaria delle Marche, Presidio Salesi, Ancona, Italy.
  • Galeazzi T; Division of Pediatrics, Department of Clinical Sciences, Azienda Ospedaliero Universitaria delle Marche, Presidio Salesi, Ancona, Italy.
  • Santori E; Division of Pediatrics, Department of Clinical Sciences, Azienda Ospedaliero Universitaria delle Marche, Presidio Salesi, Ancona, Italy.
  • Cordiali R; Division of Pediatrics, Department of Clinical Sciences, Azienda Ospedaliero Universitaria delle Marche, Presidio Salesi, Ancona, Italy.
  • Dardis A; Regional Coordinator Centre for Rare Diseases, Academic Hospital "Santa Maria della Misericordia", Udine, Italy.
  • Catassi C; Division of Pediatrics, Department of Clinical Sciences, Azienda Ospedaliero Universitaria delle Marche, Presidio Salesi, Ancona, Italy.
  • Boccieri E; The Division of Pediatric Gastroenterology and Nutrition and Center for Celiac Research, Mass General Hospital for Children, Boston, Massachusetts, USA.
  • Galaverna F; Department of Pediatric Hematology and Oncology and Cell and Gene Therapy, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy.
  • Locatelli F; Department of Pediatric Hematology and Oncology and Cell and Gene Therapy, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy.
Am J Med Genet A ; 191(7): 1948-1952, 2023 07.
Article in En | MEDLINE | ID: mdl-37045799
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cell Transplantation / Alpha-Mannosidosis Type of study: Diagnostic_studies / Screening_studies Limits: Humans / Infant Language: En Journal: Am J Med Genet A Journal subject: GENETICA MEDICA Year: 2023 Type: Article Affiliation country: Italy
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cell Transplantation / Alpha-Mannosidosis Type of study: Diagnostic_studies / Screening_studies Limits: Humans / Infant Language: En Journal: Am J Med Genet A Journal subject: GENETICA MEDICA Year: 2023 Type: Article Affiliation country: Italy