ROVI Pharma Industrial Services S.A.

The objective of this study was to evaluate the safety, efficacy, and tolerability of long-term dosing of Ataluren 10 mg/kg, 10 mg/kg, and 20 mg/kg in nmDMD subjects who wished to continue treatment after completing a Phase III study (PTC124-GD-041-DMD). The safety profile of ataluren will be described based on the type, frequency, severity, timing, and relationship to ataluren of all adverse events (AEs), laboratory abnormalities, vital signs, electrocardiogram (ECG), echocardiogram, and physical examination findings. The efficacy of ataluren will be evaluated based on change from baseline in upper limb function (PUL), DMD upper limb patient-reported outcome measures (PROMs), spirometry, loss of ambulation ability (LoA), and 6-minute walk test (6MWT).