Work

Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in subjects with mucopolysaccharidosis type VII < 5 years old

Year: 2020

Type: article

Study design: Observational Study

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2019.11.239

Authors: Heather Lau, David Viskochil, Pranoot Tanpaiboon, Antonio Gonzalez-Meneses Lopez, Esmeralda Martins, Julie Taylor, Tricia Cimms, Betsy C. Malkus, Ting Chang, Lin Zhang, Deborah Marsden, Agnieszka Jurecka

Institutions: New York University, University of Utah, Children's National, Hospital Universitario Virgen del Rocío, Universidad de Sevilla, Hospital de Santo António, Centro Hospitalar do Porto, Ultragenyx Pharmaceutical (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: closed

OpenAlex ID: W3003868843

FWCI:

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