Treatment with Ataluren for Duchene Muscular Dystrophy

Autor: Mercuri, E, Muntoni, F, Osorio, An, Tulinius, M, Buccella, F, Morgenroth, Lp, Gordish-Dressman, H, Jiang, J, Trifillis, P, Zhu, J, Kristensen, A, Santos, Cl, Henricson, Ek, Mcdonald, Cm, Desguerre, I, Bernert, G, Gosk-Tomek, M, Ille, A, Kellersmann, A, Weiss, S, Pilshofer, V, Balintovà, Z, Danhofer, P, Fabulovà, P, Jurıkovà, L, Fuchsovà, P, Haberlovà, J, Laffargue, F, Sarret, C, Pontier, B, Bellance, R, Sarrazin, E, Sabouraud, P, Magot, A, Mercier, S, Péréon, Y, Cuisset, J-M, Coopman-Degryse, S, Enaud, E, Jacquemont, M-L, Perville, A, Renouil, M, Trommsdorff, V, Verheulpen, D, Fontaine-Carbonnel, S, Vuillerot, C, Peudenier, S, Ropars, J, Audic, F, Chabrol, B, Chabrier, S, Gousse, G, Lagrue, E, Aragon, K, Barnerias, C, Brande, Lv, De Lucia, S, Gidaro, T, Seferian, A, Servais, L, Laugel, V, Espil-Taris, C, Mecili, H, Raffo, E, Ragot-Mandry, S, Borrell, S, Kirschner, J, Gangfuss, A, Henrich, M, Kolbel, H, Schara, U, Sponemann, N, Temme, E, Seeger, J, Hirsch, A, Denecke, J, Johannsen, J, Neu, A, Osinski, D, Rugner, S, Schussler, S, Trollmann, R, Kaindl, A, Schneider, Jb, Stoltenburg, C, Weiss, C, Schreiber, G, Hahn, A, Grzybowski, M, Pavlidou, E, Pavlou, E, Dobner, S, Liptai, Z, Dor, T, Brogna, C, Catteruccia, M, D’Amico, A, Pane, E, Bello, L, Pegoraro, E, Semplicini, C, Albamonte, E, Baranello, G, Comi, G, Govoni, A, Lerario, A, Magri, F, Masson, R, Mauri, E, Sansone, V, Brusa, C, Mongini, T, Ricci, F, Vacchetti, M, Bruno, C, Paniucci, C, Pedemonte, M, Giannotta, M, Pini, A, Messina, S, Sframeli, M, Vita, Gl, Vita, G, Ruggiero, L, Santoro, L, Craiu, D, Motoescu, C, Sandu, C, Teleanu, R, Vasile, D, Hughes, I, Childs, A-M, Alhaswani, Z, Roper, H, Parasuraman, D, Degoede, C, Gowda, V, Manzur, A, Munot, P, Sarkokzy, A, Charlesworth, C, Lemon, J, Turner, L, Spinty, S, Dubrovsky, A, Kornberg, A, Ryan, M, Webster, R, Biggar, Wd, Mcadam, Lc, Mah, Jh, Kolski, H, Vishwanathan, V, Chidambaranathan, S, Nevo, Y, Gorni, K, Carlo, J, Abresch, Rt, Joyce, Nc, Cnaan, A, Leshner, R, Tesi-Rocha, C, Thangarajh, M, Duong, T, Clemens, Pr, Abdel-Hamid, H, Connolly, Am, Pestronk, A, Teasley, J, Harper, A, Bertorini, Te, Kuntz, N, Driscoll, S, Day, Jw, Karachunski, P, Lotze, T.

Publikováno v: Pediatric Neurology Briefs
Journal of Comparative Effectiveness Research

Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c8c3d73c52768050ba6c0561d601a50f
http://europepmc.org/articles/PMC7718099

Comparison of SpO(2) to PaO(2) based markers of lung disease severity for children with acute lung injury.

Autor: Khemani RG, Thomas NJ, Venkatachalam V, Scimeme JP, Berutti T, Schneider JB, Ross PA, Willson DF, Hall MW, Newth CJ, Pediatric Acute Lung Injury and Sepsis Network Investigators (PALISI)

Publikováno v: Critical Care Medicine; Apr2012, Vol. 40 Issue 4, p1309-1316, 8p

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Blood pressure variability during pediatric cardiac surgery is associated with acute kidney injury.

Autor: Fishbein JE; Department of Pediatrics, Cohen Children's Medical Center, 269-01 76th Avenue, New Hyde Park, NY, 11040, USA., Barone M; Department of Pediatrics, Division of Nephrology, Cohen Children's Medical Center, 269-01 76th Avenue, New Hyde Park, NY, 11040, USA., Schneider JB; Pediatric Critical Care Medicine, Cohen Children's Medical Center, 269-01 76th Avenue, New Hyde Park, NY, 11040, USA., Meyer DB; Cardiothoracic Surgery, Cohen Children's Medical Center, 269-01 76th Avenue, New Hyde Park, NY, 11040, USA., Hagen J; Department of Anesthesiology, Cohen Children's Medical Center, 269-01 76th Avenue, New Hyde Park, NY, 11040, USA., Bakar A; Pediatric Critical Care Medicine, Cohen Children's Medical Center, 269-01 76th Avenue, New Hyde Park, NY, 11040, USA., Grammatikopoulos K; Department of Pediatrics, Division of Nephrology, Cohen Children's Medical Center, 269-01 76th Avenue, New Hyde Park, NY, 11040, USA., Sethna CB; Department of Pediatrics, Division of Nephrology, Cohen Children's Medical Center, 269-01 76th Avenue, New Hyde Park, NY, 11040, USA. csethna@northwell.edu.; Division of Pediatric Nephrology, 269-01 76th Avenue, New Hyde Park, NY, 11040, USA. csethna@northwell.edu.

Publikováno v: Pediatric nephrology (Berlin, Germany) [Pediatr Nephrol] 2022 Apr; Vol. 37 (4), pp. 871-879. Date of Electronic Publication: 2021 Aug 26.