Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy

BACKGROUND: Mucopolysaccharidosis IVA (MPS IVA, also called Morquio A syndrome) is caused by a deficiency of N‐acetylglucosamine‐6‐sulfate sulfatase (GALNS) and results in skeletal dysplasia symptoms such as short stature and abnormal gait. Treatments include enzyme replacement therapy (ERT) and hem...

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Detalles Bibliográficos
Autores principales: Chen, Hui, Khan, Shaukat, Celik, Betul, Suzuki, Yasuyuki, Ago, Yasuhiko, Tomatsu, Shunji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8606213/
https://www.ncbi.nlm.nih.gov/pubmed/34623762
http://dx.doi.org/10.1002/mgg3.1806

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8606213/
https://www.ncbi.nlm.nih.gov/pubmed/34623762
http://dx.doi.org/10.1002/mgg3.1806

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