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Phase III rugonersen in Angelman syndrome
2025-523729-16-00Phase 3Authorised
- Sponsor
- OHB Pediatrics
- Start
- Oct 2026
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 13
01
Overview
Phase 3, randomised, double-blind trial of rugonersen in Angelman syndrome (AS) (2 arms), sponsored by OHB Pediatrics. Primary endpoint: Change from baseline in Bayley-4 cognition and/or expressive communication raw scores without caregiver input at Week 56. Planned start Oct 2026; status: authorised.
02
Drugs
rugonersen
03
Conditions
Angelman syndrome (AS)
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