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Haas M, Vlcek V, Balabanov P, Salmonson T, Bakchine S, Markey G, Weise M, Schlosser-Weber G, Brohmann H, Yerro CP, Mendizabal MR, Stoyanova-Beninska V, Hillege HL. European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene. Neuromuscular Disord. 2015;25(1):5-13.
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Weise, M.7
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Brohmann, H.9
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Mendizabal, M.R.11
Stoyanova-Beninska, V.12
Hillege, H.L.13
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Ataluren treatment of patients with nonsense mutation dystrophinopathy
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PTC124-GD-007-DMDSTUDYGROUP
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Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, Connolly AM, Day JW, Flanigan KM, Goemans N, Jones KJ, Mercuri E, Quinlivan R, Renfroe JB, Russman B, Ryan MM, Tulinius M, Voit T, Moore SA, Lee Sweeney H, Abresch RT, Coleman KL, Eagle M, Florence J, Gappmaier E, Glanzman AM, Henricson E, Barth J, Elfring GL, Reha A, Spiegel RJ, O'donnell MW, Peltz SW, Mcdonald CM, PTC124-GD-007-DMDSTUDYGROUP. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve. 2014;50(4):477-87.
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Campbell, C.5
Comi, G.P.6
Connolly, A.M.7
Day, J.W.8
Flanigan, K.M.9
Goemans, N.10
Jones, K.J.11
Mercuri, E.12
Quinlivan, R.13
Renfroe, J.B.14
Russman, B.15
Ryan, M.M.16
Tulinius, M.17
Voit, T.18
Moore, S.A.19
Lee Sweeney, H.20
Abresch, R.T.21
Coleman, K.L.22
Eagle, M.23
Florence, J.24
Gappmaier, E.25
Glanzman, A.M.26
Henricson, E.27
Barth, J.28
Elfring, G.L.29
Reha, A.30
Spiegel, R.J.31
O'Donnell, M.W.32
Peltz, S.W.33
Mcdonald, C.M.34
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