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RO7248824 (rugonersen) safety, tolerability, pharmacokinetics, and pharmacodynamics study in Angelman syndrome
NL-OMON59607Phase 1Completed
- Sponsor
- Roche
- Start
- Jan 2021
- 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
- 1
01
Overview
Phase 1, open-label, single-arm trial of rugonersen in Angelman syndrome (AS), sponsored by Roche. Primary endpoints: Frequency and severity of adverse events, serious adverse events, and treatment discontinuations due to adverse events, Frequency of abnormal laboratory findings and 2 more. Started Jan 2021; status: completed.
02
Drugs
rugonersen
03
Conditions
Angelman syndrome (AS)
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