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

NCT07474298 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

NOT_YET_RECRUITING

Status

1

Enrollment

OTHER

Sponsor class

Conditions

Schuurs-Hoeijmakers Syndrome

Interventions

DRUG: nL-PACS1-001

Sponsor

n-Lorem Foundation

Collaborators

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