Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA.

Background And Aims: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study.

A comparative experimental investigation of dynamic viscosity of ZrO/DW and SiC/DW nanofluids: Characterization, rheological behavior, and development of new correlation.

In general, A nanofluid is a substance in which solids and fluids are mixed. The nano-powder of zirconium oxide (ZrO) and silicon carbide (SiC) was dispersed into the distilled water (DW) using the widely adopted two-step technique. A Brookfield viscometer was used to measure the viscosity of the nanoparticles of ZrO/DW and SiC/DW, where the temperature ranged between 20 and 60 °C and different solid volume fractions of 0.

Predictors of 6-year event-free survival in Alagille syndrome patients treated with maralixibat, an ileal bile acid transporter inhibitor.

Background And Aims: Refractory pruritus and other complications of cholestasis are indications for liver transplantation (LT) in patients with Alagille syndrome (ALGS). We evaluated predictors of event-free survival and transplant-free survival in patients with ALGS treated with maralixibat (MRX), an ileal bile acid transporter inhibitor.

Approach And Results: We assessed patients with ALGS from 3 clinical trials of MRX with up to 6 years of follow-up.

Safety, tolerability, and efficacy of maralixibat in adults with primary sclerosing cholangitis: Open-label pilot study.

Background: Primary sclerosing cholangitis (PSC) is frequently associated with pruritus, which significantly impairs quality of life. Maralixibat is a selective ileal bile acid transporter (IBAT) inhibitor that lowers circulating bile acid (BA) levels and reduces pruritus in cholestatic liver diseases. This is the first proof-of-concept study of IBAT inhibition in PSC.