Your browser doesn't support javascript.
loading
One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency.
Diaz, George A; Jones, Simon A; Scarpa, Maurizio; Mengel, Karl Eugen; Giugliani, Roberto; Guffon, Nathalie; Batsu, Isabela; Fraser, Patricia A; Li, Jing; Zhang, Qi; Ortemann-Renon, Catherine.
Afiliação
  • Diaz GA; Icahn School of Medicine at Mount Sinai, New York, NY, USA. george.diaz@mssm.edu.
  • Jones SA; St Mary's Hospital, Manchester University Foundation Trust, University of Manchester, Manchester, UK.
  • Scarpa M; Regional Coordinating Center for Rare Diseases, University Hospital Udine, Udine, Italy.
  • Mengel KE; Clinical Science for LSD, SpinCS, Hochheim, Germany.
  • Giugliani R; Dept Genetics, UFRGS, Medical Genetics Clinical Research Group, HCPA, and INAGEMP, Porto Alegre, Brazil.
  • Guffon N; Reference Center for Inherited Metabolic Disorders, Femme Mère Enfant Hospital, Lyon, France.
  • Batsu I; Sanofi, Bridgewater, NJ, USA.
  • Fraser PA; Sanofi Genzyme, Inc, Cambridge, MA, USA.
  • Li J; Sanofi, Bridgewater, NJ, USA.
  • Zhang Q; Sanofi, Bridgewater, NJ, USA.
  • Ortemann-Renon C; Sanofi, Bridgewater, NJ, USA.
Genet Med ; 23(8): 1543-1550, 2021 08.
Article em En | MEDLINE | ID: mdl-33875845
Assuntos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Niemann-Pick Tipo A Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adolescent / Child / Child, preschool / Humans / Infant Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Niemann-Pick Tipo A Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adolescent / Child / Child, preschool / Humans / Infant Idioma: En Ano de publicação: 2021 Tipo de documento: Article