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ArticleNature medicine2025

The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial.

Jörg F Hipp et al.PubMed ↗Publisher ↗

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25 papers cite it

2025
2026
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The trial

Full record →Abstract, authors, funding and every citing paper · PMID 40646322