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| 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 |
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- Mercuri, E.
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- Bernert, G.
- Gosk-Tomek, M.
- Ille, A.
- Kellersmann, A.
- Weiss, S.
- Pilshofer, V.
- Balintova, Z.
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- Jurikova, L.
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- Bellance, R.
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- Aragon, K.
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- Brande, L. V.
- 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, J. B.
- 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, M.
- 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, G.
- Ruggiero, L.
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- Childs, A. M.
- Alhaswani, Z.
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- 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, W. D.
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- Abresch, R. T.
- Joyce, N. C.
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- Thangarajh, M.
- Duong, T.
- Clemens, P. R.
- Abdel-Hamid, H.
- Connolly, A. M.
- Pestronk, A.
- Teasley, J.
- Harper, A.
- Bertorini, T. E.
- Kuntz, N.
- Driscoll, S.
- Day, J. W.
- Karachunski, P.
- Lotze, T.
- Stride,, Stride
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