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Upregulation of acid ceramidase contributes to tumor progression in tuberous sclerosis complex.
Astrinidis A, Li C, Zhang EY, Zhao X, Zhao S, Guo M, Olatoke T, Mattam U, Huang R, Zhang AG, Pitstick L, Kopras EJ, Gupta N, Jandarov R, Smith EP, Fugate E, Lindquist D, Markiewski MM, Karbowniczek M, Wikenheiser-Brokamp KA, Setchell KDR, McCormack FX, Xu Y, Yu JJ. Astrinidis A, et al. Among authors: setchell kdr. JCI Insight. 2023 May 8;8(9):e166850. doi: 10.1172/jci.insight.166850. JCI Insight. 2023. PMID: 36927688 Free PMC article.
In vivo, 17a significantly decreased the growth of TSC2-null cell-derived mouse xenografts and short-term lung colonization by TSC2-null cells. Combined rapamycin and 17a treatment synergistically inhibited renal cystadenoma growth in Tsc2+/- mice, consistent with increase …
In vivo, 17a significantly decreased the growth of TSC2-null cell-derived mouse xenografts and short-term lung colonization by TSC2-n …
Comprehensive and long-term outcomes of enzyme replacement therapy followed by stem cell transplantation in children with Gaucher disease type 1 and 3.
Anurathapan U, Tim-Aroon T, Zhang W, Sanpote W, Wongrungsri S, Khunin N, Chutipongtanate S, Chirdkiatgumchai V, Ngiwsara L, Jaovisidha S, Khongkraparn A, Pakakasama S, Svasti J, Setchell KDR, Wattanasirichaigoon D, Hongeng S. Anurathapan U, et al. Among authors: setchell kdr. Pediatr Blood Cancer. 2023 Mar;70(3):e30149. doi: 10.1002/pbc.30149. Epub 2022 Dec 23. Pediatr Blood Cancer. 2023. PMID: 36562549
METHODS: We explored combination therapy of ERT followed by hematopoietic stem cell transplantation (HSCT) and its long-term outcomes in patients with GD type 3 (GD3). RESULTS: Four patients with GD3 and one with GD type 1 (GD1) underwent HSCT. ...
METHODS: We explored combination therapy of ERT followed by hematopoietic stem cell transplantation (HSCT) and its long-term outcomes …
Maralixibat for the treatment of PFIC: Long-term, IBAT inhibition in an open-label, Phase 2 study.
Loomes KM, Squires RH, Kelly D, Rajwal S, Soufi N, Lachaux A, Jankowska I, Mack C, Setchell KDR, Karthikeyan P, Kennedy C, Dorenbaum A, Desai NK, Garner W, Jaecklin T, Vig P, Miethke A, Thompson RJ. Loomes KM, et al. Among authors: setchell kdr. Hepatol Commun. 2022 Sep;6(9):2379-2390. doi: 10.1002/hep4.1980. Epub 2022 May 4. Hepatol Commun. 2022. PMID: 35507739 Free PMC article. Clinical Trial.
Reliance on surgical interventions, including liver transplantation, highlights the unmet therapeutic need. INDIGO was an open-label, Phase 2, international, long-term study to assess the efficacy and safety of maralixibat in children with FIC1 or BSEP deficiencies. ...Pat …
Reliance on surgical interventions, including liver transplantation, highlights the unmet therapeutic need. INDIGO was an open-label, Phase …
Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study.
Gonzales E, Hardikar W, Stormon M, Baker A, Hierro L, Gliwicz D, Lacaille F, Lachaux A, Sturm E, Setchell KDR, Kennedy C, Dorenbaum A, Steinmetz J, Desai NK, Wardle AJ, Garner W, Vig P, Jaecklin T, Sokal EM, Jacquemin E. Gonzales E, et al. Among authors: setchell kdr. Lancet. 2021 Oct 30;398(10311):1581-1592. doi: 10.1016/S0140-6736(21)01256-3. Epub 2021 Oct 28. Lancet. 2021. PMID: 34755627 Clinical Trial.
Subsequently, all participants received open-label maralixibat until week 48. During the long-term extension (204 weeks reported), doses were increased up to 380 mug/kg twice per day. ...
Subsequently, all participants received open-label maralixibat until week 48. During the long-term extension (204 weeks reported), do …
Abnormal Bilirubin Metabolism in Patients With Sodium Taurocholate Cotransporting Polypeptide Deficiency.
Yan YY, Wang MX, Gong JY, Liu LL, Setchell KDR, Xie XB, Wang NL, Li W, Wang JS. Yan YY, et al. Among authors: setchell kdr. J Pediatr Gastroenterol Nutr. 2020 Nov;71(5):e138-e141. doi: 10.1097/MPG.0000000000002862. J Pediatr Gastroenterol Nutr. 2020. PMID: 33093374
The serum direct bilirubin level in NTCP-deficient patients was significantly higher than age- and sex-matched controls even after the neonatal cholestasis stage (2.85 1.50 vs 1.49 0.70 mumol/L, P = 0.00008). No growth delay or other severe long-term clinical consequences …
The serum direct bilirubin level in NTCP-deficient patients was significantly higher than age- and sex-matched controls even after the neona …
Model-Informed Bayesian Estimation Improves the Prediction of Morphine Exposure in Neonates and Infants.
Euteneuer JC, Mizuno T, Fukuda T, Zhao J, Setchell KDR, Muglia LJ, Vinks AA. Euteneuer JC, et al. Among authors: setchell kdr. Ther Drug Monit. 2020 Oct;42(5):778-786. doi: 10.1097/FTD.0000000000000763. Ther Drug Monit. 2020. PMID: 32427759 Free PMC article.
BACKGROUND: Pain control in infants is an important clinical concern, with potential long-term adverse neurodevelopmental effects. Intravenous morphine is routinely administered for postoperative pain management; however, its dose-concentration-response relationship in neo …
BACKGROUND: Pain control in infants is an important clinical concern, with potential long-term adverse neurodevelopmental effects. In …
Open-label Phase 3 Continuation Study of Cholic Acid in Patients With Inborn Errors of Bile Acid Synthesis.
Heubi JE, Setchell KDR. Heubi JE, et al. Among authors: setchell kdr. J Pediatr Gastroenterol Nutr. 2020 Apr;70(4):423-429. doi: 10.1097/MPG.0000000000002618. J Pediatr Gastroenterol Nutr. 2020. PMID: 31899729 Clinical Trial.
CONCLUSION: Oral cholic acid provides a safe and efficacious short- and long-term therapy for patients with BASD....
CONCLUSION: Oral cholic acid provides a safe and efficacious short- and long-term therapy for patients with BASD....
Electronic Health Record-Embedded Decision Support Platform for Morphine Precision Dosing in Neonates.
Vinks AA, Punt NC, Menke F, Kirkendall E, Butler D, Duggan TJ, Cortezzo DE, Kiger S, Dietrich T, Spencer P, Keefer R, Setchell KDR, Zhao J, Euteneuer JC, Mizuno T, Dufendach KR. Vinks AA, et al. Among authors: setchell kdr. Clin Pharmacol Ther. 2020 Jan;107(1):186-194. doi: 10.1002/cpt.1684. Epub 2019 Dec 11. Clin Pharmacol Ther. 2020. PMID: 31618453 Free PMC article.
In intravenous form, it is administered as continuous infusions and intermittent injections, mostly based on empirically established protocols. Inadequate pain control in neonates can cause long-term adverse consequences; however, providing appropriate individualized morph …
In intravenous form, it is administered as continuous infusions and intermittent injections, mostly based on empirically established protoco …
delta4-3-oxosteroid-5beta-reductase deficiency: Responses to oral bile acid therapy and long-term outcomes.
Zhang MH, Setchell KD, Zhao J, Gong JY, Lu Y, Wang JS. Zhang MH, et al. Among authors: setchell kd. World J Gastroenterol. 2019 Feb 21;25(7):859-869. doi: 10.3748/wjg.v25.i7.859. World J Gastroenterol. 2019. PMID: 30809085 Free PMC article.
CONCLUSION: The primary bile acid CDCA is effective in treating AKR1D1 deficiency but the therapeutic dose requires individualized optimization. UDCA is not recommended for long-term management....
CONCLUSION: The primary bile acid CDCA is effective in treating AKR1D1 deficiency but the therapeutic dose requires individualized optimizat …
Long-Term Cholic Acid Therapy in Zellweger Spectrum Disorders.
Heubi JE, Setchell KDR, Bove KE. Heubi JE, et al. Among authors: setchell kdr. Case Rep Gastroenterol. 2018 Jun 28;12(2):360-372. doi: 10.1159/000490095. eCollection 2018 May-Aug. Case Rep Gastroenterol. 2018. PMID: 30057520 Free PMC article.
Oral treatment with primary bile acid, cholic acid (CA), inhibits formation of hepatotoxic C(27)-bile acids by restoring normal physiologic feedback inhibition on bile acid synthesis. We present the long-term CA treatment and liver-related outcomes for 3 pediatric patients …
Oral treatment with primary bile acid, cholic acid (CA), inhibits formation of hepatotoxic C(27)-bile acids by restoring normal physiologic …
30 results