New hope for angelman syndrome: drug trial targets brain function
Disease control
Not yet recruiting
This study tests a drug called rugonersen, given via spinal injection, to see if it can improve thinking and communication skills in people with Angelman syndrome. The trial includes 165 children and adults aged 1 to 50. Researchers will compare the drug against a sham procedure …
Phase: PHASE3 • Sponsor: OHB Pediatrics Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:03 UTC