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...

Description complète

Enregistré dans:
Détails bibliographiques
Auteurs principaux: Seung Hoon Lee, Hwa Young Kim, Tae-Joon Cho, Hyoungmin Kim, Jung Min Ko
Format: Artigo
Langue:Inglês
Publié: Elsevier 2022-06-01
Collection:Molecular Genetics and Metabolism Reports
Sujets:
Treatment outcome
Elosulfase alfa
Enzyme replacement therapy
Morquio A syndrome
Mucopolysaccharidosis type IVA
Korean
Accès en ligne:http://www.sciencedirect.com/science/article/pii/S2214426922000295
Tags: Ajouter un tag
Pas de tags, Soyez le premier à ajouter un tag!

Documents similaires