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Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry.
Muenzer, Joseph; Jones, Simon A; Tylki-Szymanska, Anna; Harmatz, Paul; Mendelsohn, Nancy J; Guffon, Nathalie; Giugliani, Roberto; Burton, Barbara K; Scarpa, Maurizio; Beck, Michael; Jangelind, Yvonne; Hernberg-Stahl, Elizabeth; Larsen, Maria Paabøl; Pulles, Tom; Whiteman, David A H.
Afiliação
  • Muenzer J; Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Jones SA; Willink Unit, Manchester Centre for Genomic Medicine, St Mary's Hospital, Manchester and Academic Health Sciences Centre, Central Manchester University Hospitals NHS Foundation Trust, Manchester, UK.
  • Tylki-Szymanska A; Department of Pediatric Nutrition and Metabolic Diseases, The Children's Memorial Health Institute, Warsaw, Poland.
  • Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.
  • Mendelsohn NJ; Genomic Medicine Program, Children's Hospitals and Clinics of Minnesota, Minneapolis, MN, USA.
  • Guffon N; Department of Pediatrics, Division of Genetics, University of Minnesota, Minneapolis, MN, USA.
  • Giugliani R; Centre de Référence des Maladies Héréditaires du Métabolisme, Hôpital Femme Mère Enfant, Bron, France.
  • Burton BK; Department of Genetics/UFRGS and INAGEMP, Medical Genetics Service/HCPA, Porto Alegre, Brazil.
  • Scarpa M; Division of Genetics, Birth Defects and Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA.
  • Beck M; Rare Disease Centre, Helios Dr Horst Schmidt Clinic, Wiesbaden, Germany.
  • Jangelind Y; Department of Women's and Children's Health, University of Padova, Padova, Italy.
  • Hernberg-Stahl E; Department of Pediatrics, University Medical Center, Johannes Gutenberg University, Mainz, Germany.
  • Larsen MP; Jangelind Consulting AB, Stockholm, Sweden.
  • Pulles T; Late Phase Solutions Europe AB, Täby, Sweden.
  • Whiteman DAH; Shire Human Genetic Therapies, Inc., 300 Shire Way HA100-310, Lexington, MA, 02421, USA.
Orphanet J Rare Dis ; 12(1): 82, 2017 05 02.
Article em En | MEDLINE | ID: mdl-28464912
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Sistema de Registros / Mucopolissacaridose II / Terapia de Reposição de Enzimas / Iduronato Sulfatase Limite: Humans Idioma: En Ano de publicação: 2017 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Sistema de Registros / Mucopolissacaridose II / Terapia de Reposição de Enzimas / Iduronato Sulfatase Limite: Humans Idioma: En Ano de publicação: 2017 Tipo de documento: Article