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. Author manuscript; available in PMC: 2021 Jun 2.
Published in final edited form as: Sci Transl Med. 2020 Dec 2;12(572):eabb5413. doi: 10.1126/scitranslmed.abb5413

Figure 4. Quantitative MRI assessment of grey matter decline in treated vs untreated CLN2 children.

Figure 4.

The reduction in the % grey matter, assessed by MRI, is shown for each treated participant (yellow circle) above the range of grey matter decline (green bar) for untreated CLN2 children matched by % grey matter. The range of the grey matter decline is derived from the data in figures S5 and S6. Treated children with grey matter decline above the range for the untreated cohort indicates a decline that is slower and outside the 95% confidence interval for untreated children. Participants V2 and V5 were the youngest trial participants and had slow rates of decline at the time of treatment such that the effect of therapy was not yet apparent. Participant V6 had only one post-treatment scan and therefore error bars could not be calculated. Participant V7 received the lower dose (2.85×1011 gc).