Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.

McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E, Clinical Evaluator Training Group, ACT DMD Study Group

Open source

DOI: 10.1016/s0140-6736(17)31611-2
Published: 2017 Sep 23
Container: Lancet (London, England)
Publisher: Not recorded
Open access: unknown

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Cite this work

BibTeX

@article{allodium:10.1016/s0140-6736-17-31611-2,
  title = {Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.},
  author = {McDonald CM and Campbell C and Torricelli RE and Finkel RS and Flanigan KM and Goemans N and Heydemann P and Kaminska A and Kirschner J and Muntoni F and Osorio AN and Schara U and Sejersen T and Shieh PB and Sweeney HL and Topaloglu H and Tulinius M and Vilchez JJ and Voit T and Wong B and Elfring G and Kroger H and Luo X and McIntosh J and Ong T and Riebling P and Souza M and Spiegel RJ and Peltz SW and Mercuri E and Clinical Evaluator Training Group and ACT DMD Study Group},
  year = {2017},
  journal = {Lancet (London, England)},
  doi = {10.1016/s0140-6736(17)31611-2},
  url = {https://doi.org/10.1016/s0140-6736(17)31611-2}
}

RIS

TY  - JOUR
TI  - Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
AU  - McDonald CM
AU  - Campbell C
AU  - Torricelli RE
AU  - Finkel RS
AU  - Flanigan KM
AU  - Goemans N
AU  - Heydemann P
AU  - Kaminska A
AU  - Kirschner J
AU  - Muntoni F
AU  - Osorio AN
AU  - Schara U
AU  - Sejersen T
AU  - Shieh PB
AU  - Sweeney HL
AU  - Topaloglu H
AU  - Tulinius M
AU  - Vilchez JJ
AU  - Voit T
AU  - Wong B
AU  - Elfring G
AU  - Kroger H
AU  - Luo X
AU  - McIntosh J
AU  - Ong T
AU  - Riebling P
AU  - Souza M
AU  - Spiegel RJ
AU  - Peltz SW
AU  - Mercuri E
AU  - Clinical Evaluator Training Group
AU  - ACT DMD Study Group
PY  - 2017
JO  - Lancet (London, England)
DO  - 10.1016/s0140-6736(17)31611-2
UR  - https://doi.org/10.1016/s0140-6736(17)31611-2
ER  -

APA

CM, M., C, C., RE, T., RS, F., KM, F., N, G., P, H., A, K., J, K., F, M., AN, O., U, S., T, S., PB, S., HL, S., H, T., M, T., JJ, V., T, V., B, W., G, E., H, K., X, L., J, M., T, O., P, R., M, S., RJ, S., SW, P., E, M., Group, C. E. T., & Group, A. D. S. (2017). Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.. Lancet (London, England). https://doi.org/10.1016/s0140-6736(17)31611-2

Source records

pubmed · retrieved 2026-09-26T07:41:20.946Z