New hope for angelman syndrome: drug trial targets brain function
NCT ID NCT07605429
First seen Jun 27, 2026 · Last updated Jul 29, 2026 · Updated 2 times
Summary
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 to measure safety and effectiveness over 56 weeks.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Rady Children's Hospital
RECRUITINGSan Diego, California, 92123, United States
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University of North Carolina at Chapel Hill School of Medicine
RECRUITINGChapel Hill, North Carolina, 27510, United States
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