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Revenue recognition Recenue recognition - Roche and SMA Foundation (Details)
$ in Thousands
1 Months Ended 12 Months Ended
Nov. 30, 2019
USD ($)
Oct. 31, 2017
USD ($)
Nov. 30, 2014
USD ($)
Jan. 31, 2014
USD ($)
Aug. 31, 2013
USD ($)
Nov. 30, 2011
USD ($)
compound
Dec. 31, 2019
USD ($)
Dec. 31, 2018
USD ($)
deliverable
Dec. 31, 2017
USD ($)
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                  
Performance obligations satisfied in current period             $ 3,900 $ 0  
Revenue             306,980 264,734 $ 194,392
Licensing And Collaboration Agreement                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                  
Additions           $ 30,000      
Performance obligations satisfied in current period $ 15,000 $ 20,000 $ 10,000 $ 7,500 $ 10,000        
Revenue             15,200 $ 200 20,300
Revenue, remaining performance obligation, amount           $ 30,000      
Collaborative Arrangement | Roche And Sma Foundation                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                  
Collaborative arrangements number of compounds in preclinical development | compound           3      
Collaborative arrangements number of significant deliverables | deliverable               2  
Research And Development Event Milestones | Licensing And Collaboration Agreement                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                  
Revenue recognition, milestone, potential achievements             135,000    
Potential net sales milestones | Licensing And Collaboration Agreement                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                  
Revenue recognition, milestone, potential achievements             325,000    
United States                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                  
Net product sales             101,000 $ 92,000 28,800
Revenue             116,676 93,694  
Non-US                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                  
Net product sales             190,300 171,000 $ 145,200
Revenue             $ 190,304 $ 171,040