Clinical Trial Update: Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuurs-Hoeijmakers Syndrome (SHMS) [Phase 1, Phase 2]

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Summary

A clinical trial is testing a personalized antisense oligonucleotide targeting PACS1 mutations in Schuurs-Hoeijmakers syndrome; the study is not yet recruiting …

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Diseases / indications

• Schuurs-hoeijmakers Syndrome

Drugs mentioned

• PACS1-targeted antisense oligonucleotide

Snippet

Sponsor: n-Lorem Foundation | Conditions: Schuurs-Hoeijmakers Syndrome | Phase 1, Phase 2 | Status: NOT_YET_RECRUITING