Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa

BACKGROUND: This post hoc subanalysis examined outcomes in adult patients with Morquio A (mucopolysaccharidosis IVA) who received enzyme replacement therapy (ERT) with elosulfase alfa over a 120-weeks period. Patients ≥18 years of age evaluated in an open-label, long-term extension study of elosulfa...

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Dades bibliogràfiques
Publicat a:Orphanet J Rare Dis
Autors principals: Hughes, D., Giugliani, R., Guffon, N., Jones, S. A., Mengel, K. E., Parini, R., Matousek, R., Hawley, S. M., Quartel, A.
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2017
Matèries:
Research
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5442692/
https://ncbi.nlm.nih.gov/pubmed/28535791
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0634-0
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