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Correction to: Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study.
Mengel, Eugen; Bembi, Bruno; Del Toro, Mireia; Deodato, Federica; Gautschi, Matthias; Grunewald, Stephanie; Grønborg, Sabine; Héron, Bénédicte; Maier, Esther M; Roubertie, Agathe; Santra, Saikat; Tylki-Szymanska, Anna; Day, Simon; Symonds, Tara; Hudgens, Stacie; Patterson, Marc C; Guldberg, Christina; Ingemann, Linda; Petersen, Nikolaj H T; Kirkegaard, Thomas; Dali, Christine Í.
Afiliação
  • Mengel E; SphinCS GmbH, Institute of Clinical Science for LSD, Hochheim, Germany. eugen.mengel@sphincs.de.
  • Bembi B; Regional Coordinator Centre for Rare Diseases, Academic Hospital Santa Maria Della Misericordia, Udine, Italy.
  • Del Toro M; Vall D'Hebron University Hospital, Barcelona, Spain.
  • Deodato F; Ospedale Pediatrico Bambino Gesù, IRCCS, 00146, Rome, Italy.
  • Gautschi M; Inselspital, University Hospital of Bern, Bern, Switzerland.
  • Grunewald S; Metabolic Department, Great Ormond Street Hospital NHS Foundation Trust, Institute for Child Health, NIHR Biomedical Research Centre UCL, London, UK.
  • Grønborg S; Centre for Inherited Metabolic Diseases, Copenhagen University Hospital (Rigshospitalet), Copenhagen, Denmark.
  • Héron B; Reference Centre for Lysosomal Disease, Trousseau University Hospital, Paris, France.
  • Maier EM; Dr. Von Hauner Children's Hospital, University of Munich, Munich, Germany.
  • Roubertie A; Institute of Neurosciences, University Hospital of Montpellier, Montpellier, France.
  • Santra S; Birmingham Children's Hospital, Birmingham, UK.
  • Tylki-Szymanska A; Children's Memorial Health Institute, Warsaw, Poland.
  • Day S; Clinical Trials Consulting and Training Limited, Buckingham, UK.
  • Symonds T; Clinical Outcomes Solutions Limited, Folkestone, UK.
  • Hudgens S; Clinical Outcomes Solutions Inc, Tucson, AZ, USA.
  • Patterson MC; Mayo Clinic Children's Center, Rochester, MN, USA.
  • Guldberg C; Orphazyme A/S, Copenhagen, Denmark.
  • Ingemann L; Orphazyme A/S, Copenhagen, Denmark.
  • Petersen NHT; Orphazyme A/S, Copenhagen, Denmark.
  • Kirkegaard T; Orphazyme A/S, Copenhagen, Denmark.
  • Dali CÍ; Orphazyme A/S, Copenhagen, Denmark.
Orphanet J Rare Dis ; 16(1): 246, 2021 Jun 01.
Article em En | MEDLINE | ID: mdl-34074315

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Etiology_studies / Observational_studies Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Alemanha

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Etiology_studies / Observational_studies Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Alemanha
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