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Impact of long-term administration of maralixibat on children with cholestasis secondary to Alagille syndrome.
Shneider BL, Spino CA, Kamath BM, Magee JC, Ignacio RV, Huang S, Horslen SP, Molleston JP, Miethke AG, Kohli R, Leung DH, Jensen MK, Loomes KM, Karpen SJ, Mack C, Rosenthal P, Squires RH, Baker A, Rajwal S, Kelly D, Sokol RJ, Thompson RJ; for ChiLDReN and UK IMAGO/IMAGINE Investigators. Shneider BL, et al. Among authors: sokol rj. Hepatol Commun. 2022 Aug;6(8):1922-1933. doi: 10.1002/hep4.1992. Epub 2022 Jun 7. Hepatol Commun. 2022. PMID: 35672955 Free PMC article.
Use of funded multicenter prospective longitudinal databases to inform clinical trials in rare diseases-Examination of cholestatic liver disease in Alagille syndrome.
Shneider BL, Kamath BM, Magee JC, Goodrich NP, Loomes KM, Ye W, Spino C, Alonso EM, Molleston JP, Bezerra JA, Wang KS, Karpen SJ, Horslen SP, Guthery SL, Rosenthal P, Squires RH, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Shneider BL, et al. Among authors: sokol rj. Hepatol Commun. 2022 Aug;6(8):1910-1921. doi: 10.1002/hep4.1970. Epub 2022 May 4. Hepatol Commun. 2022. PMID: 35506349 Free PMC article.
The conduct of long-term conventional randomized clinical trials in rare diseases is very difficult, making evidenced-based drug development problematic. ...
The conduct of long-term conventional randomized clinical trials in rare diseases is very difficult, making evidenced-based drug deve …
Incidence, Impact and Treatment of Ongoing CMV Infection in Patients with Biliary Atresia in Four European Centres.
Fischler B, Czubkowski P, Dezsofi A, Liliemark U, Socha P, Sokol RJ, Svensson JF, Davenport M. Fischler B, et al. Among authors: sokol rj. J Clin Med. 2022 Feb 11;11(4):945. doi: 10.3390/jcm11040945. J Clin Med. 2022. PMID: 35207217 Free PMC article.
In the largest centre, CMV infection was more common in non-Caucasians, and CMV infected patients had poorer long-term survival with native liver than CMV negative patients (p = 0.0001). ...We conclude that ongoing CMV infection at the time of KPE occurs in a significant p …
In the largest centre, CMV infection was more common in non-Caucasians, and CMV infected patients had poorer long-term survival with …
Neonatal Cholestasis: Updates on Diagnostics, Therapeutics, and Prevention.
Feldman AG, Sokol RJ. Feldman AG, et al. Among authors: sokol rj. Neoreviews. 2021 Dec 1;22(12):e819-e836. doi: 10.1542/neo.22-12-e819. Neoreviews. 2021. PMID: 34850148 Free PMC article. Review.
New lipid emulsion formulations, coupled with preventive measures to decrease central line-associated bloodstream infections, have resulted in lower rates of cholestasis and liver disease in infants and children receiving long-term parental nutrition. Unfortunately, little …
New lipid emulsion formulations, coupled with preventive measures to decrease central line-associated bloodstream infections, have resulted …
Impact of Genotype, Serum Bile Acids, and Surgical Biliary Diversion on Native Liver Survival in FIC1 Deficiency.
van Wessel DBE, Thompson RJ, Gonzales E, Jankowska I, Shneider BL, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipiński P, Czubkowski P, Rock N, Shagrani M, Broering D, Algoufi T, Mazhar N, Nicastro E, Kelly D, Nebbia G, Arnell H, Fischler B, Hulscher JBF, Serranti D, Arikan C, Debray D, Lacaille F, Goncalves C, Hierro L, Muñoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsőfi A, Luigi Calvo P, Krebs-Schmitt D, Hartleif S, van der Woerd WL, Wang JS, Li LT, Durmaz Ö, Kerkar N, Hørby Jørgensen M, Fischer R, Jimenez-Rivera C, Alam S, Cananzi M, Laverdure N, Targa Ferreira C, Ordonez F, Wang H, Sency V, Mo Kim K, Chen HL, Carvalho E, Fabre A, Quintero Bernabeu J, Alonso EM, Sokol RJ, Suchy FJ, Loomes KM, McKiernan PJ, Rosenthal P, Turmelle Y, Rao GS, Horslen S, Kamath BM, Rogalidou M, Karnsakul WW, Hansen B, Verkade HJ; Natural Course and Prognosis of PFIC and Effect of Biliary Diversion Consortium. van Wessel DBE, et al. Among authors: sokol rj. Hepatology. 2021 Aug;74(2):892-906. doi: 10.1002/hep.31787. Epub 2021 Jul 13. Hepatology. 2021. PMID: 33666275 Free PMC article.
The rarity of FIC1 deficiency has largely prevented a detailed analysis of its natural history, effects of predicted protein truncating mutations (PPTMs), and possible associations of serum bile acid (sBA) concentrations and surgical biliary diversion (SBD) with long-term …
The rarity of FIC1 deficiency has largely prevented a detailed analysis of its natural history, effects of predicted protein truncating muta …
Intestinal failure-associated liver disease (IFALD): insights into pathogenesis and advances in management.
Lee WS, Chew KS, Ng RT, Kasmi KE, Sokol RJ. Lee WS, et al. Among authors: sokol rj. Hepatol Int. 2020 May;14(3):305-316. doi: 10.1007/s12072-020-10048-8. Epub 2020 Apr 30. Hepatol Int. 2020. PMID: 32356227 Review.
Reduction in the amount of SO-ILEs or replacement with non-SO-ILEs has been shown to reverse IFALD although safety and long-term effectiveness have not been studied. Medical and surgical modalities to increase intestinal adaptation, advance enteral feedings, and prevent ce …
Reduction in the amount of SO-ILEs or replacement with non-SO-ILEs has been shown to reverse IFALD although safety and long-term effe …
New Insights Into Intestinal Failure-Associated Liver Disease in Children.
Khalaf RT, Sokol RJ. Khalaf RT, et al. Among authors: sokol rj. Hepatology. 2020 Apr;71(4):1486-1498. doi: 10.1002/hep.31152. Epub 2020 Mar 18. Hepatology. 2020. PMID: 32003009 Free PMC article. Review.
Development of intestinal failure-associated liver disease (IFALD) is a common complication of long-term parenteral nutrition (PN) in children and adults. The molecular and cellular mechanisms and the phases of IFALD are now being delineated. ...
Development of intestinal failure-associated liver disease (IFALD) is a common complication of long-term parenteral nutrition (PN) in …
Risk of Expectant Management and Optimal Timing of Delivery in Low-Risk Term Pregnancies: A Population-Based Study.
Vilchez G, Nazeer S, Kumar K, Warren M, Dai J, Sokol RJ. Vilchez G, et al. Among authors: sokol rj. Am J Perinatol. 2018 Feb;35(3):262-270. doi: 10.1055/s-0037-1607042. Epub 2017 Sep 22. Am J Perinatol. 2018. PMID: 28938501
Whether expectant management risks outweigh the benefits of awaiting 39 weeks has not been studied. We analyze the risks of expectant management at term and the optimal timing for delivery. STUDY DESIGN: All U.S. nonanomalous singleton term deliveries in 2013 were s …
Whether expectant management risks outweigh the benefits of awaiting 39 weeks has not been studied. We analyze the risks of expectant manage …
Contemporary analysis of maternal and neonatal morbidity after uterine rupture: A nationwide population-based study.
Vilchez G, Dai J, Kumar K, Lagos M, Sokol RJ. Vilchez G, et al. Among authors: sokol rj. J Obstet Gynaecol Res. 2017 May;43(5):834-838. doi: 10.1111/jog.13300. Epub 2017 Feb 11. J Obstet Gynaecol Res. 2017. PMID: 28188975
Neonates are at sizeable risk for neonatal intensive care unit admission and seizures, recognized markers of long-term neurobehavioral abnormality. Uterine rupture remains a major risk for mothers and babies....
Neonates are at sizeable risk for neonatal intensive care unit admission and seizures, recognized markers of long-term neurobehaviora …
Total Serum Bilirubin within 3 Months of Hepatoportoenterostomy Predicts Short-Term Outcomes in Biliary Atresia.
Shneider BL, Magee JC, Karpen SJ, Rand EB, Narkewicz MR, Bass LM, Schwarz K, Whitington PF, Bezerra JA, Kerkar N, Haber B, Rosenthal P, Turmelle YP, Molleston JP, Murray KF, Ng VL, Wang KS, Romero R, Squires RH, Arnon R, Sherker AH, Moore J, Ye W, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Shneider BL, et al. Among authors: sokol rj. J Pediatr. 2016 Mar;170:211-7.e1-2. doi: 10.1016/j.jpeds.2015.11.058. Epub 2015 Dec 24. J Pediatr. 2016. PMID: 26725209 Free PMC article.
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