Work

Design and rationale of the LYS-SAF302 gene therapy study in mucopolysaccharidosis type IIIA (MPS IIIA) children

Year: 2019

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2018.12.283

Authors: Sophie Olivier, Samantha Parker, Anupam Chakrapani, Ronald Crystal, Lisa Emrick, Benedicte Heron, Nicole Muschol, Raymond Wang, Chester Whitley, Frits Wijburg, Lucie Rouch

Institutions: Great Ormond Street Hospital, Weill Cornell Medicine, Hôpital Armand-Trousseau, Children's Hospital of Orange County, University of Minnesota, Amsterdam UMC Location University of Amsterdam

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology (medical)

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W2912949308

FWCI:

Cites:

Cited by: 2

Related to: 10