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Observational
Opportunity to Reach Individuals With Genetic Dyslipidemia During Infancy and the Newborn Period to Find Familial Hypercholesterolemia
The goal of this clinical trial is to identify different types of Familial Hypercholesterolemia (FH) in infants and newborns. Participants will: * undergo a c…
Heterozygous Familial Hypercholesterolemia (HeFH)Homozygous Familial Hypercholesterolemia (HoFH)Familial Hypercholesterolemia
University of Wisconsin, MadisonNCT07470723
Phase 1
A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias
This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are ref…
CardiovascularMetabolic DiseaseDyslipidemias+7 more
CRISPR Therapeutics AGNCT07491172