Elosulfase alfa enzyme replacement therapy attenuates disease progression in a non-ambulatory Japanese patient with Morquio A syndrome (case report)

Enzyme replacement therapy (ERT) with elosulfase alfa is the only approved therapy in Japan for patients with Morquio A syndrome, a lysosomal storage disorder inherited in an autosomal recessive fashion. The experience with ERT in severely affected, non-ambulatory patients has not been reported in p...

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Detalles Bibliográficos
Autores principales: Hiramatsu, Misako, Nakamura, Kimitoshi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2017
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5604956/
https://www.ncbi.nlm.nih.gov/pubmed/28971020
http://dx.doi.org/10.1016/j.ymgmr.2017.09.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5604956/
https://www.ncbi.nlm.nih.gov/pubmed/28971020
http://dx.doi.org/10.1016/j.ymgmr.2017.09.001

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