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. 2019 Feb 7;9:1633. doi: 10.1038/s41598-018-38208-9

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© The Author(s) 2019

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view- a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.

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In vivo efficacy of LDN-2014 in an intermediate Smn^2B/− mouse model. LDN-2014 (5 mg/kg), 76-series analog (5 mg/kg) or vehicle (DMSO) injections were administered by IP q.a.d. (A) Kaplan-Meier survival curve of untreated SMA mice (n = 6) in comparison to vehicle (n = 17), LDN-2014 (n = 16), and 76-analog (n = 16) treated cohorts. (B) Summary table showing the average survival and Log-rank Mantel-Cox test “p” values for the various comparison. (C) Body weight measurements of untreated SMA mice (n = 6) in comparison to vehicle (n = 16), LDN-2014 (n = 16) and 76-analog (n = 16). (D) Body weight expressed as percentage weight gain from “birth-to-peak”, from all treated groups. Statistical significance is represented by “*” where p ≤ 0.05; “**”p ≤ 0.01; “***”p ≤ 0.001; “****”p ≤ 0.001; ^“ns”p > 0.05. Data expressed as S.E.M.