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Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa.
Mitchell, John J; Burton, Barbara K; Bober, Michael B; Campeau, Philippe M; Cohen, Shelda; Dosenovic, Sara; Ellaway, Carolyn; Bhattacharya, Kaustuv; Guffon, Nathalie; Hinds, David; Lail, Alice; Lin, Shuan-Pei; Magner, Martin; Raiman, Julian; Schwartz-Sagi, Liat; Stepien, Karolina M.
Afiliación
  • Mitchell JJ; McGill University Health Centre, Montreal, Quebec, Canada. Electronic address: John.mitchell@muhc.mcgill.ca.
  • Burton BK; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA. Electronic address: bburton@luriechildrens.org.
  • Bober MB; Nemours/Alfred I. DuPont Hospital for Children, Wilmington, DE, USA. Electronic address: michael.bober@nemours.org.
  • Campeau PM; CHU Sainte-Justine Research Center, Montreal, Quebec, Canada. Electronic address: p.campeau@umontreal.ca.
  • Cohen S; BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: shelda.cohen@bmrn.com.
  • Dosenovic S; BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: sara.dosenovic@bmrn.com.
  • Ellaway C; Sydney Children's Hospital Network, Sydney, NSW, Australia. Electronic address: carolyn.ellaway@health.nsw.gov.au.
  • Bhattacharya K; Sydney Children's Hospital Network, Sydney, NSW, Australia. Electronic address: kaustuv.bhattacharya@health.nsw.gov.au.
  • Guffon N; Reference Centre of Inherited Metabolic Disease, HCL Hospital, Lyon, France. Electronic address: nathalie.guffon-fouilhoux@chu-lyon.fr.
  • Hinds D; BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: David.Hinds@bmrn.com.
  • Lail A; BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: alice.lail@bmrn.com.
  • Lin SP; MacKay Children's Hospital, Taipei, Taiwan.
  • Magner M; Department of Pediatrics and Inherited Metabolic Disorders, General University Hospital and First Faculty of Medicine, Charles University, Prague, Czech Republic. Electronic address: martin.magner@vfn.cz.
  • Raiman J; Birmingham Children's Hospital, Birmingham, UK. Electronic address: julian.raiman@nhs.net.
  • Schwartz-Sagi L; BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: Liat.SchwartzSagi@bmrn.com.
  • Stepien KM; Salford Royal NHS Foundation Trust, Salford, UK. Electronic address: karolina.stepien@nca.nhs.uk.
Mol Genet Metab ; 137(1-2): 164-172, 2022.
Article en En | MEDLINE | ID: mdl-36087504
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Mucopolisacaridosis IV Tipo de estudio: Clinical_trials / Diagnostic_studies / Observational_studies / Prognostic_studies Límite: Child / Humans Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2022 Tipo del documento: Article
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Mucopolisacaridosis IV Tipo de estudio: Clinical_trials / Diagnostic_studies / Observational_studies / Prognostic_studies Límite: Child / Humans Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2022 Tipo del documento: Article