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. 2022 Jun 17;28(7):1381–1389. doi: 10.1038/s41591-022-01866-4

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© The Author(s) 2022

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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Fig. 1 — a, Milestones achieved (visit month identified). Months calculated as days/30. Only the first observed instance of a milestone is included in this figure. ^aBayley Scales gross motor subtest item #26: child sits alone without support for at least 30 seconds. ^bBayley Scales gross motor subtest item #40: child stands alone. Child stands alone for at least 3 seconds after you release his or her hands. ^cBayley Scales gross motor subtest item #43: child walks alone. Child takes at least five steps independently, displaying coordination and balance. According to the WHO-MGRS windows for normal development, the 99th percentile (that is, upper bound of normal development) of sitting and walking without support was 279 days and 534 days, respectively. b, Kaplan–Meier plot for event-free survival in the SPR1NT two-copy (blue line) and PNCR (red line) cohorts. n = 4 males and n = 10 females; mean (s.d.) age at dosing, 20.6 (7.9) days.