Extended Data Fig. 1: Change in N-acetyl-aspartic acid (NAA) concentrations in the brain, measured by magnetic resonance spectroscopy after MYR-101 treatment.

Panel a. Mean change in NAA across the occipital region for individual participants (average of right and left occipital voxels), measured by MRS. Population fit (i.e., mean line for the entire population) is shown in red. For reference, normal age-matched NAA concentrations in the occipital lobe range from 4-7 mM. Panel b. individual NAA values for each participant, including participant for whom pre-treatment values were available; in those participants, NAA slope was positive leading up to gene therapy and negative afterwards, as previously described².