GlobalResearch and clinical trials
Rugonersen found safe in a phase 1 study in Angelman syndrome
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
A phase 1 study of a drug called rugonersen was carried out with 61 children with Angelman syndrome. The children were aged between 1 and 12. The drug aims to switch on the intact copy of the gene that stays silent. The study's main goal, safety, was met. Signs of improvement were seen in brain waves and some skills.
What does it mean for patients and families?
This is an early-stage study, and the drug is not yet approved. Larger studies are needed to confirm the effect. You can consult your child neurologist about taking part in studies.
Source
Scientific paper: The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial. Nature Medicine, 2025. DOI: 10.1038/s41591-025-03784-7 · PubMed 40646322
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Angelman syndrome
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.