Dystrophies et atrophie musculaire, la thérapie génique avance
▻https://www.osservatoriomalattierare.it/ricerca-scientifica/13085-distrofie-e-atrofie-muscolari-la-terapia-genica-avanz
« Un projet américain étudie l’utilisation de cette stratégie thérapeutique dans sept maladies différentes, grâce à la précieuse contribution d’associations telles que GFB Onlus »
First Duchenne Patient Dosed in Microdystrophin Gene Therapy!
▻http://community.parentprojectmd.org/m/blogpost?id=1187424%3ABlogPost%3A245554
Our purpose in awarding this grant was to open and accelerate the field of gene therapy – a strategy that seemed impossible as a potential treatment for Duchenne back in the 1990s. If successful, it will provide systemic delivery of a microdystrophin construct that targets both skeletal and cardiac muscle achieved through one IV injection. The FDA has approved the protocol and now we have the first child dosed.