Fig. 1.

WSiMD study demographics. The average age of participants enrolled in the trial was 35 years (range 18 to 60 years). A total of twenty patients were enrolled (7 females and 13 males). Ambulatory status was determined using Vignos lower extremity score. 9 patients were ambulatory, 6 were non-ambulatory, and 5 were considered transitioning to non-ambulatory. Dosing determined by weight: < 70 kg treated with 1.0 mg/kg/week and > 70 kg treated with 0.75 mg/kg/week. Dosing occurred weekly on Monday nights, after dinner, between 7–9 pm. The average weekly dose was 62 mg, with a range of 45 to 85 mg.