Work

Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome

Year: 2021

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2021.03.013

Authors: Shih-hsin Kan, Ibrahim Elsharkawi, Steven Q. Le, Heather Prill, Linley Mangini, Jonathan D. Cooper, Roger Lawrence, Mark S. Sands, Brett E. Crawford, Patricia I. Dickson

Institutions: Children's Hospital of Orange County, The Lundquist Institute, Harbor–UCLA Medical Center, Washington University in St. Louis, University of California, Los Angeles, BioMarin (United States)

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

Funders: BioMarin Pharmaceutical, National Institutes of Health, National Institute of General Medical Sciences, National Institute of Neurological Disorders and Stroke

Awards: G5388065200, Recombinant alpha-N-acetylglucosaminidase with improved mannose 6-phosphorylation for treatment of Sanfilippo B syndrome, Next Generation Treatment for Krabbe Disease, Phenotypic Effects of Brain-Directed Enzyme Therapy for Sanfilippo B Syndrome, UCLA Medical Genetics Training Program

OpenAlex ID: W3152426144

FWCI: 0.628

Cites: 38

Cited by: 8

Related to: 10