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Comment
. 2019 Nov;36(11):2982-2985.
doi: 10.1007/s12325-019-01088-0. Epub 2019 Sep 11.

Response to: Alfred Sandrock, Wildon Farwell. Letter to the Editor, Comparisons Between Separately Conducted Clinical Trials: Letter to the Editor Regarding Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guérin A, et al. Adv Ther (2019) 36(5):1164-76. doi:10.1007/s12325-019-00923-8

Omar Dabbous  1 , Benit Maru  1 , Jeroen P Jansen  2 , Maria Lorenzi  2 , Martin Cloutier  3 , Annie Guérin  3 , Irina Pivneva  3 , Eric Q Wu  4 , Ramesh Arjunji  1 , Douglas Feltner  1 , Douglas M Sproule  5
Affiliations
Comment

Response to: Alfred Sandrock, Wildon Farwell. Letter to the Editor, Comparisons Between Separately Conducted Clinical Trials: Letter to the Editor Regarding Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guérin A, et al. Adv Ther (2019) 36(5):1164-76. doi:10.1007/s12325-019-00923-8

Omar Dabbous et al. Adv Ther. 2019 Nov.
No abstract available

Keywords: AVXS-101; Indirect treatment comparison; Neuroscience; Nusinersen; Onasemnogene abeparvovec; Spinal muscular atrophy type 1 (SMA type 1).

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References

    1. Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014;83(9):810–817. doi: 10.1212/WNL.0000000000000741. - DOI - PMC - PubMed
    1. Wadman RI, Stam M, Gijzen M, et al. Association of motor milestones, SMN2 copy and outcome in spinal muscular atrophy types 0–4. J Neurol Neurosurg Psychiatry. 2017;88(4):365–367. doi: 10.1136/jnnp-2016-314292. - DOI - PubMed
    1. Kolb SJ, Coffey CS, Yankey JW, et al. Natural history of infantile-onset spinal muscular atrophy. Ann Neurol. 2017;82(6):883–891. doi: 10.1002/ana.25101. - DOI - PMC - PubMed
    1. De Sanctis R, Pane M, Coratti G, et al. Clinical phenotypes and trajectories of disease progression in type 1 spinal muscular atrophy. Neuromuscul Disord. 2018;28(1):24–28. doi: 10.1016/j.nmd.2017.09.015. - DOI - PubMed
    1. De Sanctis R, Coratti G, Pasternak A, et al. Developmental milestones in type I spinal muscular atrophy. Neuromuscul Disord. 2016;26(11):754–759. doi: 10.1016/j.nmd.2016.10.002. - DOI - PMC - PubMed