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Clinical trial

An Open-label Single Center Study of an Experimental Antisense Oligonucleotide Treatment of a Participant With Schuurs-Hoeijmakers Syndrome Due to PACS1 Genetic Mutation

NCT07474298 is registered in ClinicalTrials.gov with n-Lorem Foundation as lead sponsor.

NCT NCT07474298
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-label Single Center Study of an Experimental Antisense Oligonucleotide Treatment of a Participant With Schuurs-Hoeijmakers Syndrome Due to PACS1 Genetic Mutation
Lead sponsor
n-Lorem Foundation
Phase
Phase 1/2
Overall status
Not yet recruiting
Start date
April 30, 2026
Completion date
April 30, 2028
Enrollment
1 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

schuurs-hoeijmakers syndrome