▽NEJM
●11/20 16:14
Nov 18, 2024Meeting of the American Heart AssociationAAV9.LAMP2B Gene Therapy for Danon DiseaseB. Greenberg and OthersIn a study involving patients with Danon disease, AAV9.LAMP2B gene therapy appeared to be safe, increased cardiac LAMP2 expression, and was associated with clinical improvement over 24 to 54 months.Listen here: NEJM at AHA — Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon D