Rugonersen treatment for Angelman syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study tests a new medicine called rugonersen for people with Angelman syndrome. The medicine is given as an injection into the fluid around the spinal cord and may help improve symptoms by targeting the genetic cause of the condition.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 1 and 50 years old
- You have a confirmed genetic diagnosis of Angelman syndrome (UBE3A gene mutation or chromosome 15 deletion)
- You weigh more than 7.5 kg (about 17 pounds)
- You can tolerate blood draws and a lumbar puncture (spinal tap) procedure under sedation
- Your epilepsy and other medications have been stable for at least 4 weeks
- You have a reliable caregiver (parent or legal guardian) who can attend appointments and complete study assessments
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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