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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Detalles Bibliográficos
Autores principales: Hughes, D., Giugliani, R., Guffon, N., Jones, S. A., Mengel, K. E., Parini, R., Matousek, R., Hawley, S. M., Quartel, A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5442692/
https://www.ncbi.nlm.nih.gov/pubmed/28535791
http://dx.doi.org/10.1186/s13023-017-0634-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5442692/
https://www.ncbi.nlm.nih.gov/pubmed/28535791
http://dx.doi.org/10.1186/s13023-017-0634-0

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