New data presented at the Child Neurology Society Annual Meeting in Montreal give families the fullest public look yet at RGX-202, an experimental one-time gene therapy for Duchenne muscular dystrophy. The pivotal Phase 3 portion of the AFFINITY DUCHENNE trial met its main goal: average levels of a key muscle protein exceeded a preset threshold 12 weeks after treatment, and 93% of boys with evaluable muscle samples individually crossed that line, NeurologyLive reported on Oct. 16.
The presentation also described two drug-related serious side effects: inflammation of the heart muscle in an 8-year-old and liver injury without symptoms in a 10-year-old. Both resolved without lasting effects, according to the abstract. The developer, REGENXBIO, first announced topline results in May, so the main finding is not new. The conference data matter because they put the safety details and study design in front of the doctors who counsel families.
