Cardiac manifestations and effects of enzyme replacement therapy for over 10 years in adults with the attenuated form of mucopolysaccharidosis type I

BACKGROUND: Mucopolysaccharidosis type I (MPS I) is a rare autosomal recessive disease caused by a deficiency of the lysosomal enzyme α-L-iduronidase. Cardiac manifestations such as valvular heart disease are associated with poor prognosis. There have been only a few reports on the effect of long-te...

Descripción completa

Detalles Bibliográficos
Autores principales: Sugiura, Kenta, Kubo, Toru, Ochi, Yuri, Baba, Yuichi, Hirota, Takayoshi, Yamasaki, Naohito, Kitaoka, Hiroaki
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7576511/
https://www.ncbi.nlm.nih.gov/pubmed/33101981
http://dx.doi.org/10.1016/j.ymgmr.2020.100662

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7576511/
https://www.ncbi.nlm.nih.gov/pubmed/33101981
http://dx.doi.org/10.1016/j.ymgmr.2020.100662

Ejemplares similares