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Revenue recognition - Collaboration and Royalty Revenue (Details)
$ in Thousands
1 Months Ended 3 Months Ended 6 Months Ended
Jan. 31, 2025
USD ($)
Jun. 30, 2025
USD ($)
Jun. 30, 2024
USD ($)
Jun. 30, 2025
USD ($)
product
Jun. 30, 2024
USD ($)
Dec. 31, 2024
USD ($)
Nov. 30, 2011
USD ($)
Revenue recognition              
Number of approved products | product       2      
Remaining potential milestones that can be achieved           $ 150,000  
Revenue   $ 178,875 $ 186,704 $ 1,354,971 $ 396,822    
Deferred revenue   9,760   9,760   $ 5,505  
Collaboration and license revenue              
Revenue recognition              
Revenue   2,941   989,172      
Royalty revenue              
Revenue recognition              
Revenue   57,605 53,183 $ 94,044 84,337    
SMA License Agreement              
Revenue recognition              
Number of approved products | product       1      
SMA License Agreement | Collaboration and license revenue              
Revenue recognition              
Revenue     0   0    
SMA License Agreement | Royalty revenue              
Revenue recognition              
Revenue   57,600 $ 53,200 $ 94,000 $ 84,300    
License and Collaboration Agreement              
Revenue recognition              
Refund of revenue related to prior collaboration arrangements       3,500      
License and Collaboration Agreement | Novartis              
Revenue recognition              
Cash proceeds from upfront payment on collaboration agreement $ 1,000,000            
Percentage share of the income (loss) generated on U.S. sales 40.00%            
Deferred revenue   7,300   7,300      
License and Collaboration Agreement | Novartis | Collaboration and license revenue              
Revenue recognition              
Revenue   2,900   992,700      
Research And Development Event Milestones | SMA License Agreement              
Revenue recognition              
Remaining potential milestones that can be achieved   0   0      
Sales Milestones | SMA License Agreement              
Revenue recognition              
Remaining potential milestones that can be achieved   $ 150,000   $ 150,000      
Maximum | License and Collaboration Agreement | Novartis              
Revenue recognition              
Potential future proceeds contingent on achievement of developmental, regulatory and sales milestones and tiered royalties $ 1,900,000            
Maximum | Research And Development Event Milestones | SMA License Agreement              
Revenue recognition              
Remaining potential milestones that can be achieved             $ 135,000
Maximum | Sales Milestones | SMA License Agreement              
Revenue recognition              
Remaining potential milestones that can be achieved             $ 325,000