The parents of children affected by SANFILIPPO Syndrome expect the start of the gene therapy trial of LYSOGENE.

Last Updated on Tuesday, 05 April 2011 09:00 Tuesday, 05 April 2011 08:51

Sanfilippo syndrome combines rarity, severity and much shortened life expectancy in affected children. For several years families are struggling and mobilizing to support research for a cure for their children. For many of them, hope has come out when the gene therapy program SANFILIPPO A was created in 2006. Supported by Alliance SANFILIPPO, AFM-Telethon and Sanfilippo Foundation (Switzerland) this program has brought together scientific, medical, toxicological, regulatory, and of course clinical expertises from the most internationally recognized teams for this type of very specific orphan disease (more details).

The program has kept all its promises and the robust body of preclinical studies (efficacy, safety) developed during the last few years is being analyzed, in final step, by the national French agency responsible for authorizing clinical trials in France.

The sponsor of this program and clinical trial is the biotechnology company LYSOGENE. Like many actors in the field of biotherapy, LYSOGENE is a small but highly specialized and innovative company whose expertise is specific of child’s rare and neurodegenerative diseases.