Sökning: WFRF:(Mah J) > Safety and effectiv...
Fältnamn | Indikatorer | Metadata |
---|---|---|
000 | 08889naa a2202449 4500 | |
001 | oai:gup.ub.gu.se/294301 | |
003 | SwePub | |
008 | 240528s2020 | |||||||||||000 ||eng| | |
024 | 7 | a https://gup.ub.gu.se/publication/2943012 URI |
024 | 7 | a https://doi.org/10.2217/cer-2019-01712 DOI |
040 | a (SwePub)gu | |
041 | a eng | |
042 | 9 SwePub | |
072 | 7 | a ref2 swepub-contenttype |
072 | 7 | a art2 swepub-publicationtype |
100 | 1 | a Mercuri, E.4 aut |
245 | 1 0 | a Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study |
264 | 1 | b Becaris Publishing Limited,c 2020 |
520 | a 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). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p <= 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731. | |
650 | 7 | a MEDICIN OCH HÄLSOVETENSKAPx Medicinska och farmaceutiska grundvetenskaperx Neurovetenskaper0 (SwePub)301052 hsv//swe |
650 | 7 | a MEDICAL AND HEALTH SCIENCESx Basic Medicinex Neurosciences0 (SwePub)301052 hsv//eng |
700 | 1 | a Muntoni, F.4 aut |
700 | 1 | a Osorio, A. N.4 aut |
700 | 1 | a Tulinius, M.4 aut |
700 | 1 | a Buccella, F.4 aut |
700 | 1 | a Morgenroth, L. P.4 aut |
700 | 1 | a Gordish-Dressman, H.4 aut |
700 | 1 | a Jiang, J.4 aut |
700 | 1 | a Trifillis, P.4 aut |
700 | 1 | a Zhu, L.4 aut |
700 | 1 | a Kristensen, A.4 aut |
700 | 1 | a Santos, C. L.4 aut |
700 | 1 | a Henricson, E. K.4 aut |
700 | 1 | a McDonald, C. M.4 aut |
700 | 1 | a Desguerre, I.4 aut |
700 | 1 | a Bernert, G.4 aut |
700 | 1 | a Gosk-Tomek, M.4 aut |
700 | 1 | a Ille, A.4 aut |
700 | 1 | a Kellersmann, A.4 aut |
700 | 1 | a Weiss, S.4 aut |
700 | 1 | a Pilshofer, V.4 aut |
700 | 1 | a Balintova, Z.4 aut |
700 | 1 | a Danhofer, P.4 aut |
700 | 1 | a Fabulova, P.4 aut |
700 | 1 | a Jurikova, L.4 aut |
700 | 1 | a Fuchsova, P.4 aut |
700 | 1 | a Haberlova, J.4 aut |
700 | 1 | a Laffargue, F.4 aut |
700 | 1 | a Sarret, C.4 aut |
700 | 1 | a Pontier, B.4 aut |
700 | 1 | a Bellance, R.4 aut |
700 | 1 | a Sarrazin, E.4 aut |
700 | 1 | a Sabouraud, P.4 aut |
700 | 1 | a Magot, A.4 aut |
700 | 1 | a Mercier, S.4 aut |
700 | 1 | a Pereon, Y.4 aut |
700 | 1 | a Cuisset, J. M.4 aut |
700 | 1 | a Coopman-Degryse, S.4 aut |
700 | 1 | a Enaud, E.4 aut |
700 | 1 | a Jacquemont, M. L.4 aut |
700 | 1 | a Perville, A.4 aut |
700 | 1 | a Renouil, M.4 aut |
700 | 1 | a Trommsdorff, V.4 aut |
700 | 1 | a Verheulpen, D.4 aut |
700 | 1 | a Fontaine-Carbonnel, S.4 aut |
700 | 1 | a Vuillerot, C.4 aut |
700 | 1 | a Peudenier, S.4 aut |
700 | 1 | a Ropars, J.4 aut |
700 | 1 | a Audic, F.4 aut |
700 | 1 | a Chabrol, B.4 aut |
700 | 1 | a Chabrier, S.4 aut |
700 | 1 | a Gousse, G.4 aut |
700 | 1 | a Lagrue, E.4 aut |
700 | 1 | a Aragon, K.4 aut |
700 | 1 | a Barnerias, C.4 aut |
700 | 1 | a Brande, L. V.4 aut |
700 | 1 | a De Lucia, S.4 aut |
700 | 1 | a Desguerre, I.4 aut |
700 | 1 | a Gidaro, T.4 aut |
700 | 1 | a Seferian, A.4 aut |
700 | 1 | a Servais, L.4 aut |
700 | 1 | a Laugel, V.4 aut |
700 | 1 | a Espil-Taris, C.4 aut |
700 | 1 | a Mecili, H.4 aut |
700 | 1 | a Raffo, E.4 aut |
700 | 1 | a Ragot-Mandry, S.4 aut |
700 | 1 | a Borrell, S.4 aut |
700 | 1 | a Kirschner, J.4 aut |
700 | 1 | a Gangfuss, A.4 aut |
700 | 1 | a Henrich, M.4 aut |
700 | 1 | a Kolbel, H.4 aut |
700 | 1 | a Schara, U.4 aut |
700 | 1 | a Sponemann, N.4 aut |
700 | 1 | a Temme, E.4 aut |
700 | 1 | a Seeger, J.4 aut |
700 | 1 | a Hirsch, A.4 aut |
700 | 1 | a Denecke, J.4 aut |
700 | 1 | a Johannsen, J.4 aut |
700 | 1 | a Neu, A.4 aut |
700 | 1 | a Osinski, D.4 aut |
700 | 1 | a Rugner, S.4 aut |
700 | 1 | a Schussler, S.4 aut |
700 | 1 | a Trollmann, R.4 aut |
700 | 1 | a Kaindl, A.4 aut |
700 | 1 | a Schneider, J. B.4 aut |
700 | 1 | a Stoltenburg, C.4 aut |
700 | 1 | a Weiss, C.4 aut |
700 | 1 | a Schreiber, G.4 aut |
700 | 1 | a Hahn, A.4 aut |
700 | 1 | a Grzybowski, M.4 aut |
700 | 1 | a Pavlidou, E.4 aut |
700 | 1 | a Pavlou, E.4 aut |
700 | 1 | a Dobner, S.4 aut |
700 | 1 | a Liptai, Z.4 aut |
700 | 1 | a Dor, T.4 aut |
700 | 1 | a Brogna, C.4 aut |
700 | 1 | a Catteruccia, M.4 aut |
700 | 1 | a D'Amico, A.4 aut |
700 | 1 | a Mercuri, E.4 aut |
700 | 1 | a Pane, M.4 aut |
700 | 1 | a Bello, L.4 aut |
700 | 1 | a Pegoraro, E.4 aut |
700 | 1 | a Semplicini, C.4 aut |
700 | 1 | a Albamonte, E.4 aut |
700 | 1 | a Baranello, G.4 aut |
700 | 1 | a Comi, G.4 aut |
700 | 1 | a Govoni, A.4 aut |
700 | 1 | a Lerario, A.4 aut |
700 | 1 | a Magri, F.4 aut |
700 | 1 | a Masson, R.4 aut |
700 | 1 | a Mauri, E.4 aut |
700 | 1 | a Sansone, V.4 aut |
700 | 1 | a Brusa, C.4 aut |
700 | 1 | a Mongini, T.4 aut |
700 | 1 | a Ricci, F.4 aut |
700 | 1 | a Vacchetti, M.4 aut |
700 | 1 | a Bruno, C.4 aut |
700 | 1 | a Paniucci, C.4 aut |
700 | 1 | a Pedemonte, M.4 aut |
700 | 1 | a Giannotta, M.4 aut |
700 | 1 | a Pini, A.4 aut |
700 | 1 | a Messina, S.4 aut |
700 | 1 | a Sframeli, M.4 aut |
700 | 1 | a Vita, G.4 aut |
700 | 1 | a Ruggiero, L.4 aut |
700 | 1 | a Santoro, L.4 aut |
700 | 1 | a Craiu, D.4 aut |
700 | 1 | a Motoescu, C.4 aut |
700 | 1 | a Sandu, C.4 aut |
700 | 1 | a Teleanu, R.4 aut |
700 | 1 | a Vasile, D.4 aut |
700 | 1 | a Tulinius, Mar,d 1953u Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics4 aut0 (Swepub:gu)xtulim |
700 | 1 | a Hughes, I.4 aut |
700 | 1 | a Childs, A. M.4 aut |
700 | 1 | a Alhaswani, Z.4 aut |
700 | 1 | a Roper, H.4 aut |
700 | 1 | a Parasuraman, D.4 aut |
700 | 1 | a DeGoede, C.4 aut |
700 | 1 | a Gowda, V.4 aut |
700 | 1 | a Manzur, A.4 aut |
700 | 1 | a Munot, P.4 aut |
700 | 1 | a Sarkokzy, A.4 aut |
700 | 1 | a Charlesworth, C.4 aut |
700 | 1 | a Lemon, J.4 aut |
700 | 1 | a Turner, L.4 aut |
700 | 1 | a Spinty, S.4 aut |
700 | 1 | a Dubrovsky, A.4 aut |
700 | 1 | a Kornberg, A.4 aut |
700 | 1 | a Ryan, M.4 aut |
700 | 1 | a Webster, R.4 aut |
700 | 1 | a Biggar, W. D.4 aut |
700 | 1 | a McAdam, L. C.4 aut |
700 | 1 | a Mah, J. K.4 aut |
700 | 1 | a Kolski, H.4 aut |
700 | 1 | a Vishwanathan, V.4 aut |
700 | 1 | a Chidambaranathan, S.4 aut |
700 | 1 | a Nevo, Y.4 aut |
700 | 1 | a Gorni, K.4 aut |
700 | 1 | a Carlo, J.4 aut |
700 | 1 | a McDonald, C. M.4 aut |
700 | 1 | a Henricson, E. K.4 aut |
700 | 1 | a Abresch, R. T.4 aut |
700 | 1 | a Joyce, N. C.4 aut |
700 | 1 | a Cnaan, A.4 aut |
700 | 1 | a Morgenroth, L. P.4 aut |
700 | 1 | a Leshner, R.4 aut |
700 | 1 | a Tesi-Rocha, C.4 aut |
700 | 1 | a Thangarajh, M.4 aut |
700 | 1 | a Duong, T.4 aut |
700 | 1 | a Clemens, P. R.4 aut |
700 | 1 | a Abdel-Hamid, H.4 aut |
700 | 1 | a Connolly, A. M.4 aut |
700 | 1 | a Pestronk, A.4 aut |
700 | 1 | a Teasley, J.4 aut |
700 | 1 | a Harper, A.4 aut |
700 | 1 | a Bertorini, T. E.4 aut |
700 | 1 | a Kuntz, N.4 aut |
700 | 1 | a Driscoll, S.4 aut |
700 | 1 | a Day, J. W.4 aut |
700 | 1 | a Karachunski, P.4 aut |
700 | 1 | a Lotze, T.4 aut |
700 | 1 | a Stride,, Stride,4 aut |
710 | 2 | a Göteborgs universitetb Institutionen för kliniska vetenskaper, Avdelningen för pediatrik4 org |
773 | 0 | t Journal of Comparative Effectiveness Researchd : Becaris Publishing Limitedg 9:5, s. 341-360q 9:5<341-360x 2042-6305x 2042-6313 |
856 | 4 | u https://doi.org/10.2217/cer-2019-0171 |
856 | 4 8 | u https://gup.ub.gu.se/publication/294301 |
856 | 4 8 | u https://doi.org/10.2217/cer-2019-0171 |
Kungliga biblioteket hanterar dina personuppgifter i enlighet med EU:s dataskyddsförordning (2018), GDPR. Läs mer om hur det funkar här.
Så här hanterar KB dina uppgifter vid användning av denna tjänst.
Kopiera och spara länken för att återkomma till aktuell vy