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Revenue recognition - Narrative (Details)
1 Months Ended 3 Months Ended 6 Months Ended 12 Months Ended
Oct. 31, 2017
USD ($)
Nov. 30, 2014
USD ($)
Jan. 31, 2014
USD ($)
Aug. 31, 2013
USD ($)
Nov. 30, 2011
USD ($)
compound
Jun. 30, 2019
USD ($)
Jun. 30, 2018
USD ($)
Jun. 30, 2019
USD ($)
segment
deliverable
Jun. 30, 2018
USD ($)
Dec. 31, 2017
USD ($)
deliverable
Dec. 31, 2018
USD ($)
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Contract with Customer, Liability           $ 15,802,000 $ 10,540,000 $ 15,802,000 $ 10,540,000 $ 11,891,000 $ 12,938,000
Number of operating segments | segment               1      
Net product revenue           85,476,000 68,170,000 $ 138,530,000 124,151,000    
Upfront cash payment               2,864,000 2,586,000    
Revenue           85,522,000 68,743,000 139,105,000 124,805,000    
Performance obligations satisfied in current period           85,476,000 68,170,000 138,530,000 124,151,000    
Licensing And Collaboration Agreement                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Upfront cash payment         $ 30,000,000.0            
Performance obligations satisfied in current period $ 20,000,000.0 $ 10,000,000 $ 7,500,000 $ 10,000,000.0              
Licensing And Collaboration Agreement | Research And Development Event Milestones                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Revenue recognition, milestone, potential achievements         135,000,000.0     87,500,000      
Licensing And Collaboration Agreement | Sales Milestones                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Revenue recognition, milestone, potential achievements         $ 325,000,000.0     325,000,000.0      
Discovery Agreements                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Revenues               0 0    
Early Stage Collaborations                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Revenues               0 0    
Early Stage Collaborations | Research And Development Event Milestones                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Revenue recognition, milestone, potential achievements               143,000,000.0      
Early Stage Collaborations | Sales Milestones                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Revenue recognition, milestone, potential achievements               252,000,000.0      
United States                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Net product revenue           27,600,000 20,300,000 45,400,000 39,500,000    
Non-US                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Net product revenue           57,800,000 47,800,000 $ 93,000,000.0 84,600,000    
Collaborative Arrangement | Roche And Sma Foundation                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Number of compounds in preclinical development | compound         3            
Number of significant deliverables | deliverable                   2  
Collaboration And Discovery Agreements                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Number of significant deliverables | deliverable               2      
Minimum | Collaboration And Discovery Agreements                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Collaborative arrangements research period for applying discovery technology               3 years      
Maximum | Collaboration And Discovery Agreements                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Collaborative arrangements research period for applying discovery technology               4 years      
Roche | Licensing And Collaboration Agreement                      
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                      
Revenues           $ 100,000 $ 100,000 $ 100,000 $ 100,000