Efficacy DataNew DMD efficacy data at 20mg/kg look favorable and reaffirm the view that there’s likely a real drug here that’s differentiated from other skippers.
Financial PerformanceThe company ended the quarter with cash, equivalents, and marketable securities of $454 mm compared to $123 mm at the end of FY22.
Safety ProfileThe absence of consistent data concerning thrombocytopenia, or anemia, or ion imbalance, is encouraging with respect to the safety profile of both ‘251 and ‘101.