U.S., Sept. 30 -- ClinicalTrials.gov registry received information related to the study (NCT07197268) titled 'Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation' on July 24.
Brief Summary: This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Bainbridge-Ropers Syndrome (BRPS) due to a pathogenic, de novo nonsense variant in ASXL3
Study Start Date: May 19
Study Type: INTERVENTIONAL
Condition:
Bainbridge-Ropers Syndrome
Intervention:
DRUG: nL-ASXL3-001
Personalized antisense oligonucleotide
Recruitment Status: ACTIVE_NOT_RECRUITING
Sponsor: n-Lorem Foundation
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