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

Abstract 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)...

Descripción completa

Guardado en:
Detalles Bibliográficos
Autores principales: Hui Chen, Shaukat Khan, Betul Celik, Yasuyuki Suzuki, Yasuhiko Ago, Shunji Tomatsu
Formato: article
Lenguaje:EN
Publicado: Wiley 2021
Materias:
Acceso en línea:https://doaj.org/article/b80043a4953a40238c94a7180891960a
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!