Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease

Autor: Baodong Sun, Suhrad G. Banugaria, Sean N. Prater, Trusha T. Patel, Keri Fredrickson, Douglas J. Ringler, Antonin de Fougerolles, Amy S. Rosenberg, Herman Waldmann, Priya S. Kishnani

Publikováno v: Molecular Genetics and Metabolism Reports, Vol 1, Iss C, Pp 446-450 (2014)

Approximately 35–40% of patients with classic infantile Pompe disease treated with enzyme replacement therapy (ERT) develop high, sustained antibody titers against the therapeutic enzyme alglucosidase alfa, which abrogates the treatment efficacy. I

Externí odkaz: https://doaj.org/article/e2218e4187334a8baee1035af47e2f37

Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT.

Autor: Suhrad G Banugaria, Sean N Prater, Trusha T Patel, Stephanie M Dearmey, Christie Milleson, Kathryn B Sheets, Deeksha S Bali, Catherine W Rehder, Julian A J Raiman, Raymond A Wang, Francois Labarthe, Joel Charrow, Paul Harmatz, Pranesh Chakraborty, Amy S Rosenberg, Priya S Kishnani

Publikováno v: PLoS ONE, Vol 8, Iss 6, p e67052 (2013)

Although enzyme replacement therapy (ERT) is a highly effective therapy, CRIM-negative (CN) infantile Pompe disease (IPD) patients typically mount a strong immune response which abrogates the efficacy of ERT, resulting in clinical decline and death.

Externí odkaz: https://doaj.org/article/18fa0e438966415a88a3734dd619fdd0

Canakinumab for the treatment of autoinflammatory very early onset- inflammatory bowel disease.

Autor: Shaul E; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States., Conrad MA; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States.; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, United States., Dawany N; Department of Biomedical and Health Informatics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States., Patel T; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States.; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, United States., Canavan MC; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States., Baccarella A; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States., Weinbrom S; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States., Aleynick D; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States., Sullivan KE; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, United States.; Division of Allergy and Immunology, The Children's Hospital of Philadelphia, Philadelphia, PA, United States., Kelsen JR; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States.; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, United States.

Publikováno v: Frontiers in immunology [Front Immunol] 2022 Sep 20; Vol. 13, pp. 972114. Date of Electronic Publication: 2022 Sep 20 (Print Publication: 2022).

Ruxolitinib: Targeted Approach for Treatment of Autoinflammatory Very Early Onset Inflammatory Bowel Disease.

Autor: Rudra S; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Shaul E; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Conrad M; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania., Patel T; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania., Moore A; Division of Clinical Pharmacy, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Dawany N; Department of Biomedical and Health Informatics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Canavan MC; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Sullivan KE; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Division of Allergy Immunology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Behrens E; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Division of Rheumatology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania., Kelsen JR; Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, The Children's Hospital of Philadelphia, Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania. Electronic address: kelsen@chop.edu.

Publikováno v: Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association [Clin Gastroenterol Hepatol] 2022 Jun; Vol. 20 (6), pp. 1408-1410.e2. Date of Electronic Publication: 2021 Jul 27.

Mucosal Invariant T cells are Diminished in Very Early-Onset Inflammatory Bowel Disease.

Autor: Dou Y; Division of Allergy Immunology., Maurer K; Division of Allergy Immunology., Conrad M; Division of Gastroenterology, Hepatology, and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA., Patel T; Division of Gastroenterology, Hepatology, and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA., Shraim R; Division of Gastroenterology, Hepatology, and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA., Sullivan KE; Division of Allergy Immunology., Kelsen J; Division of Gastroenterology, Hepatology, and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA.

Publikováno v: Journal of pediatric gastroenterology and nutrition [J Pediatr Gastroenterol Nutr] 2021 Oct 01; Vol. 73 (4), pp. 529-536.