Clinical characteristics and effects of enzyme replacement therapy with elosulfase alfa in Korean patients with mucopolysaccharidosis type IVA.

Lee, Seung Hoon; Kim, Hwa Young; Cho, Tae-Joon; Kim, Hyoungmin; Ko, Jung Min

Lee, Seung Hoon; Kim, Hwa Young; Cho, Tae-Joon; Kim, Hyoungmin; Ko, Jung Min.

Affiliation

Lee SH; Department of Pediatrics, Seoul National University College of Medicine, Republic of Korea.
Kim HY; Department of Pediatrics, Seoul National University College of Medicine, Republic of Korea.
Cho TJ; Department of Orthopaedics, Seoul National University College of Medicine, Republic of Korea.
Kim H; Department of Orthopaedics, Seoul National University College of Medicine, Republic of Korea.
Ko JM; Department of Pediatrics, Seoul National University College of Medicine, Republic of Korea.

Mol Genet Metab Rep ; 31: 100869, 2022 Jun.

Article in En | MEDLINE | ID: mdl-35782601

Key words

6MWT, 6-min walk test; ERT, enzyme replacement therapy; Elosulfase alfa; Enzyme replacement therapy; FIM, (Functional Independence Measure); GAGs, glycosaminoglycans; GALNS, N-acetylgalactosamine-6-sulfatase; Korean; MPS IVA, Mucopolysaccharidosis type IVA; Morquio A syndrome; Mucopolysaccharidosis type IVA; SDS, standard deviation score; SDS_K, standard deviation score for height based on Korean National Growth Charts; SDS_M, standard deviation score for height based on untreated MPS IVA growth reference; Treatment outcome

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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Genet Metab Rep Year: 2022 Document type: Article Country of publication:

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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Genet Metab Rep Year: 2022 Document type: Article Country of publication: