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Page 1
Trial of Erythropoietin for Hypoxic-Ischemic Encephalopathy in Newborns.
Wu YW, Comstock BA, Gonzalez FF, Mayock DE, Goodman AM, Maitre NL, Chang T, Van Meurs KP, Lampland AL, Bendel-Stenzel E, Mathur AM, Wu TW, Riley D, Mietzsch U, Chalak L, Flibotte J, Weitkamp JH, Ahmad KA, Yanowitz TD, Baserga M, Poindexter BB, Rogers EE, Lowe JR, Kuban KCK, O'Shea TM, Wisnowski JL, McKinstry RC, Bluml S, Bonifacio S, Benninger KL, Rao R, Smyser CD, Sokol GM, Merhar S, Schreiber MD, Glass HC, Heagerty PJ, Juul SE; HEAL Consortium. Wu YW, et al. N Engl J Med. 2022 Jul 14;387(2):148-159. doi: 10.1056/NEJMoa2119660. N Engl J Med. 2022. PMID: 35830641 Free PMC article. Clinical Trial.
Alzheimer's drugs, APPlication for Down syndrome?
Sokol DK, Lahiri DK. Sokol DK, et al. Ageing Res Rev. 2024 Apr;96:102281. doi: 10.1016/j.arr.2024.102281. Epub 2024 Mar 19. Ageing Res Rev. 2024. PMID: 38513771 Review.
Treatments for this AD in DS simply do not. New drug therapies for AD, e.g., Lecanemab, are monoclonal antibodies designed to clear amyloid plaques composed of Abeta. ...
Treatments for this AD in DS simply do not. New drug therapies for AD, e.g., Lecanemab, are monoclonal antibodies designed to clear a …
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. Hepatology. 2021 Aug;74(2):892-906. doi: 10.1002/hep.31787. Epub 2021 Jul 13. Hepatology. 2021. PMID: 33666275 Free PMC article.
Patients were categorized according to the number of PPTMs (i.e., splice site, frameshift due to deletion or insertion, nonsense, duplication), FIC1-A (n = 67; no PPTMs), FIC1-B (n = 29; one PPTM), or FIC1-C (n = 34; two PPTMs). ...
Patients were categorized according to the number of PPTMs (i.e., splice site, frameshift due to deletion or insertion, nonsense, dup …
Risk of variceal hemorrhage and pretransplant mortality in children with biliary atresia.
Bass LM, Ye W, Hawthorne K, Leung DH, Murray KF, Molleston JP, Romero R, Karpen S, Rosenthal P, Loomes KM, Wang KS, Squires RH, Miethke A, Ng VL, Horslen S, Kyle Jensen M, Sokol RJ, Magee JC, Shneider BL; ChiLDReN. Bass LM, et al. Hepatology. 2022 Sep;76(3):712-726. doi: 10.1002/hep.32451. Epub 2022 Apr 18. Hepatology. 2022. PMID: 35271743 Free PMC article.
Cumulative incidence of VH and transplant-free survival was compared based on features of portal hypertension (e.g., splenomegaly, thrombocytopenia) and clinical parameters at baseline in each cohort (PROBE: 1.5 to 4.5 months after hepatoportoenterostomy [HPE]; BASIC: at e …
Cumulative incidence of VH and transplant-free survival was compared based on features of portal hypertension (e.g., splenomegaly, th …
Genetic and Congenital Anomalies in Infants With Hypoxic-Ischemic Encephalopathy.
Morell AS, Monsell SE, Cornet MC, Wisnowski JL, McKinstry RC, Mathur AM, Li Y, Glass HC, Gonzalez FF, Mayock DE, Benninger KL, Van Meurs KP, Lampland AL, Wu TW, Riley D, Mietzsch U, Chalak L, Flibotte J, Weitkamp JH, Ahmad KA, Yanowitz TD, Baserga M, Merhar S, Rao R, Sokol GM, Comstock BA, Heagerty PJ, Juul SE, Wu YW. Morell AS, et al. Pediatr Neurol. 2024 May;154:44-50. doi: 10.1016/j.pediatrneurol.2024.02.007. Epub 2024 Feb 20. Pediatr Neurol. 2024. PMID: 38518503
Infants with known genetic or congenital anomalies were excluded. The primary outcome, i.e., death or neurodevelopmental impairment (NDI), was determined at age two years by a standardized neurological examination, Bayley Scales of Infant Development, Third Edition (BSID-I …
Infants with known genetic or congenital anomalies were excluded. The primary outcome, i.e., death or neurodevelopmental impairment ( …
Total serum bilirubin predicts fat-soluble vitamin deficiency better than serum bile acids in infants with biliary atresia.
Venkat VL, Shneider BL, Magee JC, Turmelle Y, Arnon R, Bezerra JA, Hertel PM, Karpen SJ, Kerkar N, Loomes KM, Molleston J, Murray KF, Ng VL, Raghunathan T, Rosenthal P, Schwartz K, Sherker AH, Sokol RJ, Teckman J, Wang K, Whitington PF, Heubi JE; Childhood Liver Disease Research and Education Network. Venkat VL, et al. J Pediatr Gastroenterol Nutr. 2014 Dec;59(6):702-7. doi: 10.1097/MPG.0000000000000547. J Pediatr Gastroenterol Nutr. 2014. PMID: 25419594 Free PMC article. Clinical Trial.