A Community-Engaged Approach to Quantifying Caregiver Preferences for the Benefits and Risks of Emerging Therapies for Duchenne Muscular Dystrophy von Peay, Holly L., MS, Hollin, Ilene, MPH, Fischer, Ryan, BA, Bridges, John F.P., PhD

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Natural history of liver disease in a large international cohort of children with Alagille syndrome: Results from the GALA study von Vandriel, Shannon M, Li, Li-Ting, She, Huiyu, Wang, Jian-She, Gilbert, Melissa A, Jankowska, Irena, Czubkowski, Piotr, Gliwicz-Miedzińska, Dorota, Gonzales, Emmanuel M, Jacquemin, Emmanuel, Bouligand, Jérôme, Spinner, Nancy B, Loomes, Kathleen M, Piccoli, David A, D'Antiga, Lorenzo, Nicastro, Emanuele, Sokal, Étienne, Demaret, Tanguy, Ebel, Noelle H, Feinstein, Jeffrey A, Fawaz, Rima, Nastasio, Silvia, Lacaille, Florence, Debray, Dominique, Arnell, Henrik, Fischler, Björn, Siew, Susan, Stormon, Michael, Karpen, Saul J, Romero, Rene, Kim, Kyung Mo, Baek, Woo Yim, Hardikar, Winita, Shankar, Sahana, Roberts, Amin J, Evans, Helen M, Jensen, M Kyle, Kavan, Marianne, Sundaram, Shikha S, Chaidez, Alexander, Karthikeyan, Palaniswamy, Sanchez, Maria Camila, Cavalieri, Maria Lorena, Verkade, Henkjan J, Lee, Way Seah, Squires, James E, Hajinicolaou, Christina, Lertudomphonwanit, Chatmanee, Fischer, Ryan T, Larson-Nath, Catherine, Mozer-Glassberg, Yael, Arikan, Cigdem, Lin, Henry C, Bernabeu, Jesus Quintero, Alam, Seema, Kelly, Deirdre A, Carvalho, Elisa, Ferreira, Cristina Targa, Indolfi, Giuseppe, Quiros-Tejeira, Ruben E, Bulut, Pinar, Calvo, Pier Luigi, Önal, Zerrin, Valentino, Pamela L, Desai, Dev M, Eshun, John, Rogalidou, Maria, Dezsőfi, Antal, Wiecek, Sabina, Nebbia, Gabriella, Pinto, Raquel Borges, Wolters, Victorien M, Tamara, María Legarda, Zizzo, Andréanne N, Garcia, Jennifer, Schwarz, Kathleen, Beretta, Marisa, Sandahl, Thomas Damgaard, Jimenez-Rivera, Carolina, Kerkar, Nanda, Brecelj, Jernej, Mujawar, Quais, Rock, Nathalie, Busoms, Cristina Molera, Karnsakul, Wikrom, Lurz, Eberhard, Santos-Silva, Ermelinda, Blondet, Niviann, Bujanda, Luis, Shah, Uzma, Thompson, Richard J, Hansen, Bettina E, Kamath, Binita M

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Levels of Interleukin-6 in Saliva, but Not Plasma, Correlate with Clinical Metrics in Huntington's Disease Patients and Healthy Control Subjects von Corey-Bloom, Jody, Fischer, Ryan S, Kim, Aeri, Snell, Chase, Parkin, Georgia M, Granger, Douglas A, Granger, Steven W, Thomas, Elizabeth A

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Adherence to laboratory testing in pediatric liver transplant recipients von Ryan, Jamie L., Dandridge, Laura M., Fischer, Ryan T.

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Whole-genome sequencing for identification of Mendelian disorders in critically ill infants: a retrospective analysis of diagnostic and clinical findings von Willig, Laurel K, MD, Petrikin, Josh E, MD, Smith, Laurie D, MD, Saunders, Carol J, PhD, Thiffault, Isabelle, PhD, Miller, Neil A, BA, Soden, Sarah E, MD, Cakici, Julie A, BSN, Herd, Suzanne M, CCRC, Twist, Greyson, MS, Noll, Aaron, MS, Creed, Mitchell, MA, Alba, Patria M, MD, Carpenter, Shannon L, MD, Clements, Mark A, MD, Fischer, Ryan T, MD, Hays, J Allyson, MD, Kilbride, Howard, Prof, McDonough, Ryan J, DO, Rosterman, Jamie L, DO, Tsai, Sarah L, MD, Zellmer, Lee, MS, Farrow, Emily G, PhD, Kingsmore, Stephen F, Prof

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Rapamycin-Conditioned Dendritic Cells Are Poor Stimulators of Allogeneic CD4+ T Cells, but Enrich for Antigen-Specific Foxp3+ T Regulatory Cells and Promote Organ Transplant Tolera... von Turnquist, Heth R, Raimondi, Giorgio, Zahorchak, Alan F, Fischer, Ryan T, Wang, Zhiliang, Thomson, Angus W

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Letter to the Editor: Oral Vancomycin Versus No Therapy for Pediatric Primary Sclerosing Cholangitis von Johnson, Kevin M., Fischer, Ryan T., Holtmann, Gerald J., Shah, Ayesha, Alrabadi, Leina, Lewindon, Peter

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Hepatic artery thrombosis and use of anticoagulants and antiplatelet agents in pediatric liver transplantation von Feldman, Keith, Heble, Jr, Daniel E, Hendrickson, Richard J, Fischer, Ryan T

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Dehydrated Hereditary Stomatocytosis Presenting as Severe Perinatal Ascites and Cholestasis von Halma, Jennifer, Petrikin, Joshua, Daniel, James F., Fischer, Ryan T.

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Use and safety of prophylactic endoscopy from a single center serving urban and rural children with portal hypertension von Slowik, Voytek, Bernardez, Anissa, Wasserkrug, Heather, Fischer, Ryan T., Daniel, James F., Grammatikopoulos, Tassos

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Uric Acid as a Potential Peripheral Biomarker for Disease Features in Huntington's Patients von Corey-Bloom, Jody, Haque, Ameera, Aboufadel, Sameer, Snell, Chase, Fischer, Ryan S, Granger, Steven W, Granger, Douglas A, Thomas, Elizabeth A

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Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA von Hansen, BE, Vandriel, SM, Vig, P, Garner, W, Mogul, DB, Loomes, KM, Piccoli, DA, Rand, EB, Jankowska, I, Czubkowski, P

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Successful Rehabilitation in Pediatric Ultrashort Small Bowel Syndrome von Infantino, Benjamin J., MD, MS, Mercer, David F., MD, PhD, FRCS(C), Hobson, Brandy D., RD, Fischer, Ryan T., MD, Gerhardt, Brandi K., RN, BAN, Grant, Wendy J., MD, Langnas, Alan N., DO, Quiros-Tejeira, Rubén E., MD

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Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy von Staunton, Hannah, Trennery, Claire, Arbuckle, Rob, Guridi, Maitea, Zhuravleva, Elena, Furlong, Pat, Fischer, Ryan, Hall, Rebecca

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Natural killer cells in liver transplantation: Can we harness the power of the immune checkpoint to promote tolerance? von Halma, Jennifer, Pierce, Stephen, McLennan, Rebecca, Bradley, Todd, Fischer, Ryan

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Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents von Landrum Peay, Holly, Fischer, Ryan, Tzeng, Janice P, Hesterlee, Sharon E, Morris, Carl, Strong Martin, Amy, Rensch, Colin, Smith, Edward, Ricotti, Valeria, Beaverson, Katherine, Wand, Hannah, Mansfield, Carol

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The effect of neuropsychiatric medication on pediatric nonalcoholic fatty liver disease von Ryan, Jamie L., Sherman, Ashley K., Heble, Daniel E., Friesen, Craig A., Daniel, James F., Fischer, Ryan T., Slowik, Voytek

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Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients von Paquin, Ryan S, Fischer, Ryan, Mansfield, Carol, Mange, Brennan, Beaverson, Katherine, Ganot, Annie, Martin, Amy Strong, Morris, Carl, Rensch, Colin, Ricotti, Valeria, Russo, Leo J, Sadosky, Alesia, Smith, Edward C, Peay, Holly L

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Design of an MRI Compatible Steerable Guide for MRI-Guided Laser Interstitial Thermal Therapy von Kim, Jin Seob, Fischer, Ryan R., Cajigas, Iahn, Ivan, Michael E., Chang, Doyoung

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Barriers to ideal outcomes after pediatric liver transplantation von Ng, Vicky Lee, Mazariegos, George V., Kelly, Beau, Horslen, Simon, McDiarmid, Sue V., Magee, John C., Loomes, Kathleen M., Fischer, Ryan T., Sundaram, Shikha S., Lai, Jennifer C., Te, Helen S., Bucuvalas, John C.

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