Work

Intracerebral administration of rAAV2/5hNAGLU vector in children with MPS IIIB: results at 30 months of a phase I/II trial

Year: 2017

Type: article

Study design: Clinical Trial

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2016.11.341

Authors: Marc Tardieu, Michel Zerah, Marie-Lise Gougeon, Jerome Ausseil, Stephanie de Bournonville, Beatrice Husson, Dimitrios Zafeiriou, GianCarlo Parenti, Philippe Bourget, Cecile Artaud, Beatrice Poirier, Noami VanVlies, Kumaran Deiva, Thomas Baugnon, Thomas Roujeau, Ronald G Crystal, Charles Richard, Jean-Michel Heard

Institutions: Université Paris-Sud, Université Paris Descartes, Institut Pasteur, Université de Picardie Jules Verne, Aristotle University of Thessaloniki, University of Amsterdam, Université de Montpellier, Cornell University, UniQure (Netherlands)

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology (medical)

Field: Medicine

Domain: Health Sciences

SDGs: Good health and well-being, Quality education

Open Access status: closed

OpenAlex ID: W2584970562

FWCI: 0.195

Cites:

Cited by: 2

Related to: 10