Clinical Trial Update: Gene Replacement Therapy for Treatment of Paediatric Patients With CTNNB1 Neurodevelopmental Syndrome [Phase 1, Phase 2]

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Summary

Ongoing Phase 1/2 gene replacement therapy trial in pediatric patients with CTNNB1 neurodevelopmental syndrome; recruitment is currently ongoing.

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Diseases / indications

• Ctnnb1 Neurodevelopmental Syndrome

Drugs mentioned

• Gene replacement therapy for CTNNB1 neurodevelopmental syndrome

Snippet

Sponsor: CTNNB1 Foundation | Conditions: CTNNB1 Neurodevelopmental Syndrome | Phase 1, Phase 2 | Status: RECRUITING