Schuurs-hoeijmakers Syndrome
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CLINICAL_TRIALS research · 2026-03-16Clinical Trial Update: Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuurs-Hoeijmakers Syndrome (SHMS) [Phase 1, Phase 2]
A clinical trial is testing a personalized antisense oligonucleotide targeting PACS1 mutations in Schuurs-Hoeijmakers syndrome; the study is…