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In the 24-week ASSERT study, treatment with odevixibat resulted in significant improvements in pruritus and reductions in bile acids versus placebo in patients with ALGS

To investigate the metabolite profile of Down syndrome using the model, we began by increasing by 50% the V max of CBS, since the gene for CBS is on chromosome 21 and is expressed at 150% of normal

Oral bioavailability studies would require dose-escalation trials measuring plasma concentrations after oral dosing versus subcutaneous dosing those trials have not been conducted or published as of 2026.

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[DOI] [PubMed] [Google Scholar] 87.Schwarzlander M., Fricker M.D., Muller C., Marty L., Brach T., Novak J., Sweetlove L.J., Hell R., Meyer A.J