Your browser doesn't support javascript.
loading
Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review.
Pineda, Mercedes; Jurícková, Katarína; Karimzadeh, Parvaneh; Kolnikova, Miriam; Malinova, Vera; Insua, Jose Luis; Velten, Christian; Kolb, Stefan A.
Afiliação
  • Pineda M; Hospital Sant Joan de Deu, Passeig de Sant Joan de Deu, 2, Esplugues de Llobregat, 08950, Barcelona, Spain. pineda@sjdhospitalbarcelona.org.
  • Jurícková K; Department of Pediatrics, Centre for Inborn Errors of Metabolism, Comenius University Children's Hospital, Bratislava, Slovakia.
  • Karimzadeh P; Department of Paediatric Neurology, Paediatric Neurology Research Centre, Shahid Beheshti University of Medical Sciences, Mofid Children Hospital, Tehran, Iran.
  • Kolnikova M; Department of Child Neurology, Comenius University Medical School and National Institute of Children's Diseases, Bratislava, Slovakia.
  • Malinova V; Department of Pediatrics and Adolescent Medicine, First Faculty of Medicine, Charles University and General University Hospital, Prague, Czech Republic.
  • Insua JL; Syntax for Science SL, Mallorca, Spain.
  • Velten C; Actelion Pharmaceuticals Ltd., Allschwil, Switzerland.
  • Kolb SA; Actelion Pharmaceuticals Ltd., Allschwil, Switzerland.
Orphanet J Rare Dis ; 14(1): 32, 2019 01 08.
Article em En | MEDLINE | ID: mdl-30732631
Assuntos
Palavras-chave
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: 1-Desoxinojirimicina / Doença de Niemann-Pick Tipo C Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Ano de publicação: 2019 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: 1-Desoxinojirimicina / Doença de Niemann-Pick Tipo C Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Ano de publicação: 2019 Tipo de documento: Article