Article
Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome.
Drug design, development and therapy - 1 Jan 2022
Lee Chung-Lin, Chuang Chih-Kuang, Chiu Huei-Ching, Tu Ru-Yi, Lo Yun-Ting, Chang Ya-Hui, Lin Shuan-Pei, Lin Hsiang-Yu
Abstract excerpt
Mucopolysaccharidosis type IVA (MPS IVA or Morquio A) is an autosomal recessive disorder and is one of the lysosomal storage diseases. Patients with MPS IVA have a striking skeletal phenotype but normal intellect. The phenotypic continuum of MPS IVA ranges from severe and rapid progress to mild and slow progress. The diagnosis of MPS IVA is usually suspected based on abnormal bone findings and dysplasia on...
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