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Gene therapy updates due at our 2019 conference

Latest gene therapy news

Dr Guiseppe Ronzitti is confirmed as a speaker at our AGSD-UK conference in October this year. A post-doctoral researcher at the University of Pierre and Marie Curie in Paris, he is a group leader at Généthon, the French AFM-Telethon funded medical research organisation which is a partner in Myocure. He was awarded a Marie Curie fellowship to work on developments towards gene therapy for GSD3.

Presentations to the Pompe and Hepatic groups

Dr Ronzitti will speak to both the GSD2 Pompe group and to the Hepatic group on “Gene Therapy Updates and Developments”. In this ever-moving field he will inform us about the most recent news.

1st FDA-approved neuromuscular gene therapy

Généthon played a decisive role in the design of both the product and the route of administration of the first FDA-approved gene therapy for a neuromuscular disease. On 24 May 2019 Zolgensma®, developed by AveXis (Novartis), was approved by the USA’s Food and Drug Administration. It is a gene therapy for the treatment of spinal muscular atrophy (SMA).

Look out for more conference info

More conference updates will follow as further speakers are confirmed.

Visit the Myocure web site. (No longer active as of October 2020.)

Learn about the basics of gene therapy

If you would like to understand more about the basics of gene therapy and gene editing, watch this informative video from The Royal Society.