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Revenue recognition - Narrative (Details)
1 Months Ended 3 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2018
USD ($)
Oct. 31, 2017
USD ($)
Nov. 30, 2014
USD ($)
Jan. 31, 2014
USD ($)
Aug. 31, 2013
USD ($)
Nov. 30, 2011
USD ($)
compound
Sep. 30, 2018
USD ($)
Sep. 30, 2018
USD ($)
deliverable
segment
Sep. 30, 2017
USD ($)
Dec. 31, 2017
deliverable
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                    
Number of operating segments | segment               1    
Net product revenue             $ 53,021,000 $ 177,172,000    
Upfront cash payment               4,706,000    
Performance obligations satisfied in current period             53,021,000 177,172,000    
Licensing And Collaboration Agreement                    
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                    
Upfront cash payment           $ 30,000,000        
Performance obligations satisfied in current period   $ 20,000,000 $ 10,000,000 $ 7,500,000 $ 10,000,000          
Revenues               200,000 $ 200,000  
Licensing And Collaboration Agreement | Research And Development Event Milestones                    
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                    
Revenue recognition, milestone, potential achievements $ 87,500,000             135,000,000    
Licensing And Collaboration Agreement | Sales Milestones                    
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                    
Revenue recognition, milestone, potential achievements               325,000,000    
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    
Early Stage Collaborations | Sales Milestones                    
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                    
Revenue recognition, milestone, potential achievements               252,000,000    
United States                    
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                    
Net product revenue             22,600,000 62,200,000    
Non-US                    
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                    
Net product revenue             $ 30,400,000 $ 115,000,000    
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