Phase 1 exploratory efficacy of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients.

Subjects

Subjects :

*THERAPEUTIC use of enzymes
*GLYCOGEN storage disease type II
*GENETIC disorders
*HEALTH impact assessment
*MEDICAL research
*THERAPEUTICS

Details

Language :

English

ISSN :

10967192

Volume :

117

Issue :

2

Database :

Academic Search Index

Journal :

Molecular Genetics & Metabolism

Publication Type :

Academic Journal

Accession number :

112948079

Full Text :

https://doi.org/10.1016/j.ymgme.2015.12.396