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Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa.
Hughes, D; Giugliani, R; Guffon, N; Jones, S A; Mengel, K E; Parini, R; Matousek, R; Hawley, S M; Quartel, A.
Afiliación
  • Hughes D; Royal Free London NHS Foundation Trust and University College London, London, UK. rmgvdah@ucl.ac.uk.
  • Giugliani R; Medical Genetics Service, Hospital de Clínicas de Porto Alegre and Department of Genetics/Universidade Federal do Rio Grande do Sul and Instituto Nacional de Genética Médica Populacional, Porto Alegre, Brazil.
  • Guffon N; Hôpital Femme Mère Enfant, Reference Centre of Inherited Metabolic Diseases, Hospices Civils de Lyon, Lyon, France.
  • Jones SA; Manchester Centre for Genomic Medicine, St Mary's Hospital, Central Manchester University Hospitals NHS Foundation Trust, University of Manchester, Manchester, UK.
  • Mengel KE; Villa Metabolica, Centre for Pediatric and Adolescent Medicine, University of Mainz Medical Center, Mainz, Germany.
  • Parini R; Fondazione Monza e Brianza per il Bambino e la sua Mamma, Azienda Ospedaliera San Gerardo, Monza, Italy.
  • Matousek R; BioMarin Pharmaceutical Inc, Novato, CA, USA.
  • Hawley SM; BioMarin Pharmaceutical Inc, Novato, CA, USA.
  • Quartel A; BioMarin Pharmaceutical Inc, Novato, CA, USA.
Orphanet J Rare Dis ; 12(1): 98, 2017 05 23.
Article en En | MEDLINE | ID: mdl-28535791
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Condroitinsulfatasas / Mucopolisacaridosis IV / Internacionalidad Tipo de estudio: Clinical_trials / Guideline / Observational_studies / Risk_factors_studies Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2017 Tipo del documento: Article
Texto completo
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Condroitinsulfatasas / Mucopolisacaridosis IV / Internacionalidad Tipo de estudio: Clinical_trials / Guideline / Observational_studies / Risk_factors_studies Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2017 Tipo del documento: Article