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Revenue recognition - Net Product Revenue (Details) - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2025
Sep. 30, 2024
Sep. 30, 2025
Sep. 30, 2024
Disaggregation of Revenue [Line Items]        
Total revenues $ 211,007 $ 196,786 $ 1,565,978 $ 593,608
United States        
Disaggregation of Revenue [Line Items]        
Total revenues 56,423 51,941 140,566 156,718
Product        
Disaggregation of Revenue [Line Items]        
Total revenues 130,956 135,421 402,711 446,245
Product | United States        
Disaggregation of Revenue [Line Items]        
Total revenues 56,423 51,941 140,566 156,718
Product | International        
Disaggregation of Revenue [Line Items]        
Total revenues 74,533 83,480 262,145 289,527
Translarna        
Disaggregation of Revenue [Line Items]        
Total revenues 50,665 72,283 196,289 246,217
Translarna | International        
Disaggregation of Revenue [Line Items]        
Total revenues 50,665 72,283 196,289 246,217
Emflaza        
Disaggregation of Revenue [Line Items]        
Total revenues 35,160 51,941 119,303 156,718
Emflaza | United States        
Disaggregation of Revenue [Line Items]        
Total revenues 35,160 51,941 119,303 156,718
Sephience        
Disaggregation of Revenue [Line Items]        
Total revenues 19,563   19,563  
Sephience | United States        
Disaggregation of Revenue [Line Items]        
Total revenues 14,365   14,365  
Sephience | International        
Disaggregation of Revenue [Line Items]        
Total revenues 5,198   5,198  
Upstaza        
Disaggregation of Revenue [Line Items]        
Total revenues 15,735   36,283 9,648
Upstaza | United States        
Disaggregation of Revenue [Line Items]        
Total revenues 6,898   6,898  
Upstaza | International        
Disaggregation of Revenue [Line Items]        
Total revenues 8,837   29,385 9,648
All other products        
Disaggregation of Revenue [Line Items]        
Total revenues 9,833 11,197 31,273 33,662
All other products | International        
Disaggregation of Revenue [Line Items]        
Total revenues $ 9,833 $ 11,197 $ 31,273 $ 33,662