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HOPE-2: open-label study of vafidemstat in adults with Phelan-McDermid syndrome
2026-525699-24-00Phase 2Authorised
- Sponsor
- Oryzon Genomics
- Start
- Sep 2026
- Modality
- Small Molecules
- Countries
- 1
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Overview
Phase 2, open-label, single-arm trial of vafidemstat in Phelan-McDermid syndrome (PMS), sponsored by Oryzon Genomics. Primary endpoints: Treatment-emergent adverse events, Serious treatment-emergent adverse events and 5 more. Planned start Sep 2026; status: authorised.
02
Drugs
vafidemstat
03
Conditions
Phelan-McDermid syndrome (PMS)
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