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Revenue recognition - Net Product Revenue (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended
Jun. 30, 2025
Jun. 30, 2024
Jun. 30, 2025
Jun. 30, 2024
Disaggregation of Revenue [Line Items]        
Total revenues $ 178,875 $ 186,704 $ 1,354,971 $ 396,822
United States        
Disaggregation of Revenue [Line Items]        
Total revenues 36,353 47,296 84,142 104,777
Product        
Disaggregation of Revenue [Line Items]        
Total revenues 118,329 133,220 271,755 310,824
Product | United States        
Disaggregation of Revenue [Line Items]        
Total revenues 36,353 47,296 84,142 104,777
Product | International        
Disaggregation of Revenue [Line Items]        
Total revenues 81,976 85,924 187,613 206,047
Translarna        
Disaggregation of Revenue [Line Items]        
Total revenues 59,470 70,350 145,624 173,934
Translarna | International        
Disaggregation of Revenue [Line Items]        
Total revenues 59,470 70,350 145,624 173,934
Emflaza        
Disaggregation of Revenue [Line Items]        
Total revenues 36,353 47,296 84,142 104,777
Emflaza | United States        
Disaggregation of Revenue [Line Items]        
Total revenues 36,353 47,296 84,142 104,777
Upstaza        
Disaggregation of Revenue [Line Items]        
Total revenues 11,889 1,589    
Upstaza | International        
Disaggregation of Revenue [Line Items]        
Total revenues 11,889 1,589    
All other products        
Disaggregation of Revenue [Line Items]        
Total revenues 10,617 13,985 41,989 32,113
All other products | International        
Disaggregation of Revenue [Line Items]        
Total revenues $ 10,617 $ 13,985 $ 41,989 $ 32,113