Publications &
Presentations
In our pursuit of scientific advancement we are committed to sharing our acquired knowledge through actively publishing our data in peer-reviewed journals and academic conferences. The focus of what we have published is on transforming the lives of people living with rare liver disease through a concentration on apical sodium-dependent bile acid transporter (ASBT) inhibition.
ASBT inhibition
An important intestinal transporter that regulates the enterohepatic circulation of bile acids and cholesterol homeostasis: the apical sodium-dependent bile acid transporter (SLC10A2/ASBT)
Xiao L, Pan G. Clin Res Hepatol Gastroenterol. 2017;41:509-515.
Role of the intestinal bile acid transporters in bile acid and drug disposition
Dawson PA. Handb Exp Pharmacol. 2011;201:169-203.
Alagille syndrome
Pediatric cholestasis: itching for an alternative to liver transplantation
Kamath BM, et al. Symposium Presentation, WCPGHAN 2021.
Outcomes of childhood cholestasis in Alagille syndrome: results of a multicenter observational study
Kamath BM, et al. Hepatol Comm. 2020;4:387-398.
Alagille syndrome and other hereditary causes of cholestasis
Hartley JL, et al. Clin Liver Dis. 2013;17:279-300.
Systematic review: the epidemiology, natural history, and burden of Alagille syndrome
Kamath BM, et al. J Pediatr Gastroenterol Nutr. 2018;67(2):148-156.
Alagille syndrome: pathogenesis, diagnosis and management
Turnpenny PD, Ellard S. Eur J Human Genetics. 2012;20:251-257.
Progressive familial intrahepatic cholestasis
Progressive familial intrahepatic cholestasis
Davit-Spraul A, et al. Orphanet J Rare Dis. 2009;4:1.
Normalization of serum bile acids after partial external biliary diversion indicates an excellent long-term outcome in children with progressive familial intrahepatic cholestasis
Schukfeh N, et al. J Pediatr Surg. 2012;47(3):501-505.
ATP8B1 and ABCB11 analysis in 62 children with normal gamma-glutamyl transferase progressive familial intrahepatic cholestasis (PFIC): phenotypic differences between PFIC1 and PFIC2 and natural history
Davit-Spraul A, et al. Hepatology. 2010;51(5):1645-1655.
Systematic review of progressive familial intrahepatic cholestasis
Baker A, et al. Clin Res Hepatol Gastroenterol. 2019;43(1):20-36.
Genotype correlates with the natural history of severe bile salt export pump deficiency
van Wessel D, et al. J Hepatol. 2020;73(1):84-93.
Patient-reported outcomes validation
Development of a novel tool to assess the impact of itching in pediatric cholestasis
Kamath BM, et al. Patient. 2018;11:69-82.
Psychometric validation of the itch reported outcome (ItchRO™) observer assessment in pediatric patients with Alagille syndrome or progressive familial intrahepatic cholestasis
Serrano D, et al. Poster Presentation, AASLD 2016.
Psychometric evaluation of the adult itch reported outcome tool, a worst itch numeric rating scale in adults with cholestatic liver disease
Foster E, et al. Poster Presentation, EASL 2020.
Maralixibat
Serum bile acid control in long-term maralixibat-treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency
Thompson R, et al. Podium Presentation, WCPGHAN 2021.
Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: an integrated analysis of short- and long-term treatment
Kamath BM, et al. Poster Presentation, WCPGHAN 2021.
Pruritus intensity is associated with cholestasis biomarkers and quality of life measures after maralixibat treatment in children with Alagille syndrome
Gonzales E, et al. Poster Presentation, AASLD 2020.
Natural variability of pruritus in Alagille syndrome; an analysis from the ICONIC study utilizing the itch reported outcome observer (ItchRO[Obs]) tool
Foster B, et al. Poster Presentation, AASLD 2020.
Serum bile acid control in long-term maralixibat-treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency
Thompson R, et al. Podium Presentation, EASL 2020.
Differential expression of bile acid subspecies with maralixibat treatment in pruritus responders with bile salt export pump deficiency
Zhao E, et al. Poster Presentation, EASL 2020.
Genotype and dose-dependent response to maralixibat in patients with bile salt export pump deficiency
Thompson R, et al. Podium Presentation, AASLD 2019.
Durability of treatment effect with long-term maralixibat in children with Alagille syndrome: 4-year safety and efficacy
Gonzales E, et al. Podium Presentation, AASLD 2019.
Safety and efficacy of maralixibat in patients with primary sclerosing cholangitis: an open-label proof-of-concept study
Bowlus C, et al. Poster Presentation, AASLD 2019.
Dose-dependent fecal bile acid excretion with apical sodium-dependent bile acid transporter inhibitors maralixibat and volixibat in a dose-ranging Phase 1 study in overweight and obese adults
Martin P, et al. Poster Presentation, AASLD 2019.
Phase 2 placebo-controlled withdrawal study of the ASBT inhibitor maralixibat in children with Alagille syndrome
Gonzales E, et al. Podium Presentation, EASL 2019.
Growth analysis in children with PFIC treated with the ASBT inhibitor maralixibat
Thompson R, et al. Podium Presentation, EASL 2019.
A randomized, controlled, Phase 2 study of maralixibat in the treatment of itching associated with primary biliary cholangitis
Mayo MJ, et al. Hepatol Commun. 2019;3(3):365-381.
Placebo-controlled randomized trial of an intestinal bile salt transport inhibitor for pruritus in Alagille syndrome
Shneider BL, et al. Hepatol Comm. 2018;2(10):1184-1198.
Phase 2 open-label efficacy and safety study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with progressive familial intrahepatic cholestasis: 48-week interim efficacy analysis
Thompson R. Podium Presentation, AASLD. 2017.
A long-term Phase 2 safety and efficacy study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with Alagille syndrome: preliminary results from the IMAGINE study
Thompson R. Podium Presentation, AASLD. 2017.
Results of ITCH, a multi-center randomized double-blind placebo-controlled trial of maralixibat, an intestinal bile salt transport inhibitor, for pruritus in Alagille syndrome
Shneider BL, et al. Podium Presentation, AASLD. 2017.
Volixibat
Safety, tolerability and efficacy of volixibat, an apical sodium-dependent bile acid transporter inhibitor, in adults with non-alcoholic steatohepatitis: 24-week interim analysis results from a Phase 2 study
Newsom P, et al. Poster Presentation, EASL. 2019.
A Phase 1 dose-ranging study assessing fecal bile acid excretion by volixibat, an apical sodium-dependent bile acid transporter inhibitor, and coadministration with loperamide
Key C, et al. Poster Presentation, AASLD. 2020.
Intrahepatic cholestasis of pregnancy
Association of adverse perinatal outcomes of intrahepatic cholestasis of pregnancy with biochemical markers: results of aggregate and individual patient data meta-analyses
Ovadia C, et al. The Lancet. 2019;393:899-909.
An integrated analysis of long-term clinical safety in maralixibat-treated participants with Alagille syndrome
Raman R, et al. Poster Presentation. EASL. 2021.
