# Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuurs-Hoeijmakers Syndrome (SHMS)

> **NCT07474298** · PHASE1,PHASE2 · NOT_YET_RECRUITING · sponsor: **n-Lorem Foundation** · enrollment: 1 (estimated)

## Conditions studied

- Schuurs-Hoeijmakers Syndrome

## Interventions

- **DRUG:** nL-PACS1-001

## Key facts

- **NCT ID:** NCT07474298
- **Lead sponsor:** n-Lorem Foundation
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** NOT_YET_RECRUITING
- **Start date:** 2026-04
- **Primary completion:** 2028-04
- **Final completion:** 2028-04
- **Target enrollment:** 1 (ESTIMATED)
- **Last updated:** 2026-03-16

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07474298

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07474298, "Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuurs-Hoeijmakers Syndrome (SHMS)". Retrieved via AI Analytics 2026-06-04 from https://api.ai-analytics.org/clinical/NCT07474298. Licensed CC0.

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