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Change history
30 March 2017
In the version of this article initially published, F.M. was credited for giving input on study design and data analysis, which he did not. The error has been corrected in the HTML and PDF versions of the article.
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Acknowledgements
F.M. is supported by the National Institute of Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London.
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F.M. was involved in the original investigator-led and independently funded phase 1 and 2A studies that led to the development of Exondys 51. F.M. has received consulting fees from PTC Therapeutics, Sarepta Therapeutics, BioMarin, Roche, Biogen, Italfarmaco, Avexis, Pfizer, Trivorsan, Catabasis and Wave Therapeutics. S.F. and S.W. are named inventors on patents describing Exondys 51 and other exon skipping compounds, which are licensed to Sarepta Therapeutics by the University of Western Australia. S.F and S.W. are consultants to Sarepta Therapeutics.
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Muntoni, F., Fletcher, S. & Wilton, S. Response to “Railroading at the FDA”. Nat Biotechnol 35, 207–209 (2017). https://doi.org/10.1038/nbt.3819
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DOI: https://doi.org/10.1038/nbt.3819
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