The first clinical trial of gene therapy for Sanfilippo

After many months of waiting for parents of children with MPS IIIA, the French drug agency (Afssaps) has authozised the completion of the clinical trial SAF-301. The sponsor is LYSOGENE, a company of biotechnology which aims at developing therapies for pathologies of the central nervous system, and the principal investigator is Prof. Marc Tardieu, neuropediatrician at Bicêtre Hospital.

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