The continuous treatment arm (n=18) showed a clinically meaningful and statistically significant difference from placebo (n=18) on 6MWD.
A new slide was shown: The 4 Stair Climb (Ascent) Grading test showed improvement in both drug treated groups compared to placebo at 24 and 48 weeks.
Drisapersen was generally well tolerated, with the majority of adverse events related to injection site and proteinuria. All patients completed the study.
The ongoing extension trial shows stabilization over three years. Since week 80, patients were on intermittent dosing and still showed stabilization. Some of these boys were almost 15 years old.