Successful prevention and stabilization of cognitive decline in Japanese patients with neuronopathic mucopolysaccharidosis type II treated by intracerebroventricular enzyme replacement therapy: Results of the Phase I/II clinical trial for two years

Subjects

Subjects :

medicine.medical_specialty
business.industry
Endocrinology, Diabetes and Metabolism
Enzyme replacement therapy
Biochemistry
Clinical trial
Endocrinology
Phase i ii
Internal medicine
Genetics
Medicine
Mucopolysaccharidosis type II
Cognitive decline
business
Molecular Biology

Details

ISSN :

10967192

Volume :

129

Database :

OpenAIRE

Journal :

Molecular Genetics and Metabolism

Accession number :

edsair.doi...........cab4a238c523da72f6d732aec8d67944

Full Text :

https://doi.org/10.1016/j.ymgme.2019.11.310