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- Safety-boosting drug may weaken gene therapy efficacy in Dravet syndrome trialEncoded Therapeutics studied a drug meant to improve the safety of gene therapy in children with Dravet syndrome, a severe genetic epilepsy. In the trial of 21 participants, most received steroids…
- Doctors link infant's brain tumor to gene therapy virus in rare caseDoctors treated 13-month-old Adam with an experimental gene therapy after his stem cell transplant failed, and the boy subsequently reached developmental milestones such as learning sign language a…