Research activity and capability in the European reference network MetabERN

by Jean-Michel Heard, Cinzia Bellettato, Corine van Lingen, Maurizio Scarpa, the MetabERN collaboration group
Published 2019-05-01

Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network

by Jean-Michel Heard, Charlotte Vrinten, Michael Schlander, Cinzia Maria Bellettato, Corine van Lingen, Maurizio Scarpa, the MetabERN collaboration group
Published 2020-01-01

Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome

by Marie-Lise Gougeon, Béatrice Poirier-Beaudouin, Jérome Ausseil, Michel Zérah, Cécile Artaud, Jean-Michel Heard, Kumaran Deiva, Marc Tardieu
Published 2021-05-01

Surrogate Cerebrospinal Fluid Biomarkers for Assessing the Efficacy of Gene Therapy in Hurler Syndrome

by Reiner F. Haseloff, Stephanie Trudel, Stephanie Trudel, Ramona Birke, Michael Schümann, Eberhard Krause, Cathy Gomila, Jean-Michel Heard, Ingolf E. Blasig, Jérôme Ausseil, Jérôme Ausseil
Published 2021-05-01

Early neurodegeneration progresses independently of microglial activation by heparan sulfate in the brain of mucopolysaccharidosis IIIB mice.

by Jérôme Ausseil, Nathalie Desmaris, Stéphanie Bigou, Ruben Attali, Sébastien Corbineau, Sandrine Vitry, Mathieu Parent, David Cheillan, Maria Fuller, Irène Maire, Marie-Thérèse Vanier, Jean-Michel Heard
Published 2008-05-01