A review of the clinical outcomes in idursulfase-treated and untreated Filipino patients with mucopolysaccharidosis type II: data from the local lysosomal storage disease registry

BACKGROUND: Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked multisystem disorder characterized by glycosaminoglycan (GAG) accumulation, caused by a deficiency of iduronate-2-sulfatase (I2S). Enzyme replacement therapy (ERT) with recombinant idursulfase (IDS), the standard of c...

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Detalles Bibliográficos
Autores principales: Racoma, Marie Julianne C., Calibag, Maria Kristina Karizza B., Cordero, Cynthia P., Abacan, Mary Ann R., Chiong, Mary Anne D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8293534/
https://www.ncbi.nlm.nih.gov/pubmed/34289859
http://dx.doi.org/10.1186/s13023-021-01875-5

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8293534/
https://www.ncbi.nlm.nih.gov/pubmed/34289859
http://dx.doi.org/10.1186/s13023-021-01875-5

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