Neurogene Gene Therapy Shows Signs of Efficacy in Small Study, But an Adverse Event Spooks Investors

https://medcitynews.com/2024/11/neurogene-gene-therapy-rett-syndrome-rare-disease-ngne/ Neurogene’s Rett syndrome gene therapy has preliminary data supporting safety and efficacy of the one-time treatment. But a late-breaking report of a serious complication in a patient who received [...]