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nL-PACS1-001 in Schuurs-Hoeijmakers Syndrome
NCT07474298Phase 2Not yet recruiting
- Sponsor
- n-Lorem Foundation
- Start
- Apr 2026
- Primary completion
- Apr 2028
- Modality
- Modified DNA Oligonucleotides, Single-strand DNA, Antisense Oligonucleotides (ASOs)
- Countries
- 1
01
Overview
A Phase 2 trial of nL-PACS1-001 in PACS1-related neurodevelopmental disorder, sponsored by n-Lorem Foundation. Planned start Apr 2026; status: not yet recruiting.
02
Drugs
nL-PACS1-001
03
Conditions
PACS1-related neurodevelopmental disorder
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