Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI: 10-year follow up (case report)

Subjects

Subjects :

Pediatrics
medicine.medical_specialty
10 year follow up
business.industry
Endocrinology, Diabetes and Metabolism
Mucopolysaccharidosis type VI
Disease progression
Enzyme replacement therapy
Biochemistry
Endocrinology
Genetics
medicine
business
Molecular Biology

Details

ISSN :

10967192

Volume :

123

Database :

OpenAIRE

Journal :

Molecular Genetics and Metabolism

Accession number :

edsair.doi...........aee57d01eb8f592d23ed51a8af067ca5

Full Text :

https://doi.org/10.1016/j.ymgme.2017.12.111