Drugs / nL-PACS1-001
last change Apr 2026 re-read 3 minutes ago

nL-PACS1-001

Antisense oligonucleotide

Developed for
Schuurs-Hoeijmakers syndrome
Investigated by
The Hospital for Sick Children · n-Lorem Foundation

Trials 1

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT07474298 Apr 2026 → Apr 2028 expected Schuurs-Hoeijmakers syndrome n-Lorem Foundation Not yet recruiting No outcome recorded

Evidence & citations 2 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as nL-PACS1-001 ClinicalTrials.gov intervention name — accepted as the source's own label NCT07474298 ↗
Modality Antisense oligonucleotide “Personalized antisense oligonucleotide” NCT07474298 ↗