ICV-administered tralesinidase alfa (BMN 250 NAGLU-IGF2) is well-tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)

Subjects

Subjects :

medicine.medical_specialty
business.industry
Endocrinology, Diabetes and Metabolism
Heparan sulfate
medicine.disease
Biochemistry
chemistry.chemical_compound
Endocrinology
chemistry
Internal medicine
Genetics
medicine
business
Molecular Biology
Sanfilippo syndrome

Details

ISSN :

10967192

Volume :

126

Database :

OpenAIRE

Journal :

Molecular Genetics and Metabolism

Accession number :

edsair.doi.dedup.....c00720e2aaed856989011fb45c4dbe0a

Full Text :

https://doi.org/10.1016/j.ymgme.2018.12.083