WFRF:(Manzur F)
Search: WFRF:(Manzur F) > (2020) > Safety and effectiv...
- 1 of 1
- Previous record
- Next record
- To hitlist
Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
- Mercuri, E. (author)
- Muntoni, F. (author)
- Osorio, A. N. (author)
- show more...
- Tulinius, M. (author)
- Buccella, F. (author)
- Morgenroth, L. P. (author)
- Gordish-Dressman, H. (author)
- Jiang, J. (author)
- Trifillis, P. (author)
- Zhu, L. (author)
- Kristensen, A. (author)
- Santos, C. L. (author)
- Henricson, E. K. (author)
- McDonald, C. M. (author)
- Desguerre, I. (author)
- Bernert, G. (author)
- Gosk-Tomek, M. (author)
- Ille, A. (author)
- Kellersmann, A. (author)
- Weiss, S. (author)
- Pilshofer, V. (author)
- Balintova, Z. (author)
- Danhofer, P. (author)
- Fabulova, P. (author)
- Jurikova, L. (author)
- Fuchsova, P. (author)
- Haberlova, J. (author)
- Laffargue, F. (author)
- Sarret, C. (author)
- Pontier, B. (author)
- Bellance, R. (author)
- Sarrazin, E. (author)
- Sabouraud, P. (author)
- Magot, A. (author)
- Mercier, S. (author)
- Pereon, Y. (author)
- Cuisset, J. M. (author)
- Coopman-Degryse, S. (author)
- Enaud, E. (author)
- Jacquemont, M. L. (author)
- Perville, A. (author)
- Renouil, M. (author)
- Trommsdorff, V. (author)
- Verheulpen, D. (author)
- Fontaine-Carbonnel, S. (author)
- Vuillerot, C. (author)
- Peudenier, S. (author)
- Ropars, J. (author)
- Audic, F. (author)
- Chabrol, B. (author)
- Chabrier, S. (author)
- Gousse, G. (author)
- Lagrue, E. (author)
- Aragon, K. (author)
- Barnerias, C. (author)
- Brande, L. V. (author)
- De Lucia, S. (author)
- Desguerre, I. (author)
- Gidaro, T. (author)
- Seferian, A. (author)
- Servais, L. (author)
- Laugel, V. (author)
- Espil-Taris, C. (author)
- Mecili, H. (author)
- Raffo, E. (author)
- Ragot-Mandry, S. (author)
- Borrell, S. (author)
- Kirschner, J. (author)
- Gangfuss, A. (author)
- Henrich, M. (author)
- Kolbel, H. (author)
- Schara, U. (author)
- Sponemann, N. (author)
- Temme, E. (author)
- Seeger, J. (author)
- Hirsch, A. (author)
- Denecke, J. (author)
- Johannsen, J. (author)
- Neu, A. (author)
- Osinski, D. (author)
- Rugner, S. (author)
- Schussler, S. (author)
- Trollmann, R. (author)
- Kaindl, A. (author)
- Schneider, J. B. (author)
- Stoltenburg, C. (author)
- Weiss, C. (author)
- Schreiber, G. (author)
- Hahn, A. (author)
- Grzybowski, M. (author)
- Pavlidou, E. (author)
- Pavlou, E. (author)
- Dobner, S. (author)
- Liptai, Z. (author)
- Dor, T. (author)
- Brogna, C. (author)
- Catteruccia, M. (author)
- D'Amico, A. (author)
- Mercuri, E. (author)
- Pane, M. (author)
- Bello, L. (author)
- Pegoraro, E. (author)
- Semplicini, C. (author)
- Albamonte, E. (author)
- Baranello, G. (author)
- Comi, G. (author)
- Govoni, A. (author)
- Lerario, A. (author)
- Magri, F. (author)
- Masson, R. (author)
- Mauri, E. (author)
- Sansone, V. (author)
- Brusa, C. (author)
- Mongini, T. (author)
- Ricci, F. (author)
- Vacchetti, M. (author)
- Bruno, C. (author)
- Paniucci, C. (author)
- Pedemonte, M. (author)
- Giannotta, M. (author)
- Pini, A. (author)
- Messina, S. (author)
- Sframeli, M. (author)
- Vita, G. (author)
- Ruggiero, L. (author)
- Santoro, L. (author)
- Craiu, D. (author)
- Motoescu, C. (author)
- Sandu, C. (author)
- Teleanu, R. (author)
- Vasile, D. (author)
- Hughes, I. (author)
- Childs, A. M. (author)
- Alhaswani, Z. (author)
- Roper, H. (author)
- Parasuraman, D. (author)
- DeGoede, C. (author)
- Gowda, V. (author)
- Manzur, A. (author)
- Munot, P. (author)
- Sarkokzy, A. (author)
- Charlesworth, C. (author)
- Lemon, J. (author)
- Turner, L. (author)
- Spinty, S. (author)
- Dubrovsky, A. (author)
- Kornberg, A. (author)
- Ryan, M. (author)
- Webster, R. (author)
- Biggar, W. D. (author)
- McAdam, L. C. (author)
- Mah, J. K. (author)
- Kolski, H. (author)
- Vishwanathan, V. (author)
- Chidambaranathan, S. (author)
- Nevo, Y. (author)
- Gorni, K. (author)
- Carlo, J. (author)
- McDonald, C. M. (author)
- Henricson, E. K. (author)
- Abresch, R. T. (author)
- Joyce, N. C. (author)
- Cnaan, A. (author)
- Morgenroth, L. P. (author)
- Leshner, R. (author)
- Tesi-Rocha, C. (author)
- Thangarajh, M. (author)
- Duong, T. (author)
- Clemens, P. R. (author)
- Abdel-Hamid, H. (author)
- Connolly, A. M. (author)
- Pestronk, A. (author)
- Teasley, J. (author)
- Harper, A. (author)
- Bertorini, T. E. (author)
- Kuntz, N. (author)
- Driscoll, S. (author)
- Day, J. W. (author)
- Karachunski, P. (author)
- Lotze, T. (author)
- Stride,, Stride, (author)
- show less...
- (creator_code:org_t)
- Becaris Publishing Limited, 2020
- 2020
- English.
- In: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
- Journal article (peer-reviewed)
Abstract
Subject headings
Close
- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Neurovetenskaper (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Neurosciences (hsv//eng)
Publication and Content Type
- ref (subject category)
- art (subject category)
Find in a library
- Journal of Comparative Effectiveness Research (Search for host publication in LIBRIS)
To the university's database
- 1 of 1
- Previous record
- Next record
- To hitlist
Find more in SwePub
- By the author/editor
- Mercuri, E.
- Muntoni, F.
- Osorio, A. N.
- Tulinius, M.
- Buccella, F.
- Morgenroth, L. P ...
- show more...
- Gordish-Dressman ...
- Jiang, J.
- Trifillis, P.
- Zhu, L.
- Kristensen, A.
- Santos, C. L.
- Henricson, E. K.
- McDonald, C. M.
- Desguerre, I.
- Bernert, G.
- Gosk-Tomek, M.
- Ille, A.
- Kellersmann, A.
- Weiss, S.
- Pilshofer, V.
- Balintova, Z.
- Danhofer, P.
- Fabulova, P.
- Jurikova, L.
- Fuchsova, P.
- Haberlova, J.
- Laffargue, F.
- Sarret, C.
- Pontier, B.
- Bellance, R.
- Sarrazin, E.
- Sabouraud, P.
- Magot, A.
- Mercier, S.
- Pereon, Y.
- Cuisset, J. M.
- Coopman-Degryse, ...
- Enaud, E.
- Jacquemont, M. L ...
- Perville, A.
- Renouil, M.
- Trommsdorff, V.
- Verheulpen, D.
- Fontaine-Carbonn ...
- Vuillerot, C.
- Peudenier, S.
- Ropars, J.
- Audic, F.
- Chabrol, B.
- Chabrier, S.
- Gousse, G.
- Lagrue, E.
- Aragon, K.
- Barnerias, C.
- 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.
- Santoro, L.
- Craiu, D.
- Motoescu, C.
- Sandu, C.
- Teleanu, R.
- Vasile, D.
- Tulinius, Mar, 1 ...
- 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, W. D.
- McAdam, L. C.
- Mah, J. K.
- Kolski, H.
- Vishwanathan, V.
- Chidambaranathan ...
- Nevo, Y.
- Gorni, K.
- Carlo, J.
- Abresch, R. T.
- Joyce, N. C.
- Cnaan, A.
- Leshner, R.
- Tesi-Rocha, C.
- 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
- show less...
- About the subject
-
- MEDICAL AND HEALTH SCIENCES
- MEDICAL AND HEAL ...
- and Basic Medicine
- and Neurosciences
- Articles in the publication
- Journal of Compa ...
- By the university
- University of Gothenburg
Search outside SwePub
- Extend your search to:
- Google Book Search
- Google Scholar
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.
- Copyright © LIBRIS - National Library Systems
- LIBRIS.kb.se
