Data Supplement

Four tables; Word document.

Files in this Data Supplement:

• E1 - Table (E) T-1. Z-scores of the individually most affected system at baseline and changes from baseline; Table (E) T-2. Visual acuity (Log Mar), nine hole peg test, and timed 8 m walking for all patients: median and 25% and 75% percentiles of raw data, and Z-scores at baseline and changes from baseline; Table (E) T-3. Z-scores of composite score: visual acuity (Log Mar), nine hole peg test, and timed 8 m walking for all patients; Table (E) T-4. Adverse event reported by the patients; Word document.