Velmanase alfa enzyme replacement therapy for alpha-mannosidosis improves patient outcomes over standard of care both in terms of clinically relevant improvement and disease stabilization

Subjects

Subjects :

medicine.medical_specialty
Standard of care
business.industry
Endocrinology, Diabetes and Metabolism
Alpha-mannosidosis
Velmanase alfa
Disease
Enzyme replacement therapy
medicine.disease
Biochemistry
Endocrinology
Internal medicine
Genetics
medicine
business
Molecular Biology

Details

ISSN :

10967192

Volume :

129

Database :

OpenAIRE

Journal :

Molecular Genetics and Metabolism

Accession number :

edsair.doi...........36805fbd4f71ee3a8c4cb67a476da104

Full Text :

https://doi.org/10.1016/j.ymgme.2019.11.171