Clinical characteristics and effects of enzyme replacement therapy with elosulfase alfa in Korean patients with mucopolysaccharidosis type IVA
Mucopolysaccharidosis type IVA (MPS IVA) is a rare autosomal recessive disorder caused by a deficiency in N-acetylgalactosamine-6-sulfatase, which results in skeletal and connective tissue abnormalities, as well as various non-skeletal manifestations. Although enzyme replacement therapy (ERT) is rec...
Main Authors: | Cho, T.-J (Author), Kim, H. (Author), Kim, H.Y (Author), Ko, J.M (Author), Lee, S.H (Author) |
---|---|
Format: | Article |
Language: | English |
Published: |
Elsevier Inc.
2022
|
Subjects: | |
Online Access: | View Fulltext in Publisher |
Similar Items
-
Role of elosulfase alfa in mucopolysaccharidosis IVA
by: Regier DS, et al.
Published: (2016-06-01) -
Elosulfase alfa in the treatment of mucopolysaccharidosis type IVA: insights from the first managed access agreement
by: Bob Stevens, et al.
Published: (2021-09-01) -
Morquio A syndrome and effect of enzyme replacement therapy in different age groups of Turkish patients: a case series
by: Sebile Kılavuz, et al.
Published: (2021-03-01) -
Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance
by: Mehmet Umut Akyol, et al.
Published: (2019-06-01) -
Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England
by: Maureen Cleary, et al.
Published: (2021-01-01)