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Revenue recognition Recenue recognition - Roche and SMA Foundation (Details)
 1 Months Ended 3 Months Ended 12 Months Ended
Dec. 31, 2018
USD ($)
Oct. 31, 2017
USD ($)
Nov. 30, 2014
USD ($)
Jan. 31, 2014
USD ($)
Aug. 31, 2013
USD ($)
Nov. 30, 2011
USD ($)
compound
Dec. 31, 2018
USD ($)
Sep. 30, 2018
USD ($)
Jun. 30, 2018
USD ($)
Mar. 31, 2018
USD ($)
Dec. 31, 2017
USD ($)
Sep. 30, 2017
USD ($)
Jun. 30, 2017
USD ($)
Mar. 31, 2017
USD ($)
Dec. 31, 2018
USD ($)
deliverable
Dec. 31, 2017
USD ($)
deliverable
Dec. 31, 2016
USD ($)
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Net Product Sales                             $ 263,005,000    
Additions                             6,417,000    
Revenue                             264,734,000 $ 194,392,000  
Net product revenue             $ 85,833,000 $ 53,021,000 $ 68,170,000 $ 55,981,000 $ 57,953,000 $ 41,780,000 $ 47,891,000 $ 26,442,000 264,734,000 194,392,000 $ 82,705,000
Licensing And Collaboration Agreement                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Performance obligations satisfied in current period   $ 20,000,000 $ 10,000,000 $ 7,500,000 $ 10,000,000                        
Additions           $ 30,000,000                      
Revenue                             200,000 $ 20,300,000 400,000
Grant [Member]                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Net product revenue                             $ 0   $ 900,000
Collaborative Arrangement [Member] | Roche And Sma Foundation [Member]                                  
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  
Collaboration And Discovery Agreements [Member]                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
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 $ 87,500,000                           $ 135,000,000    
Research And Development Event Milestones | Early Stage Collaborations                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Revenue Recognition, Milestone, Potential Achievements                             143,000,000    
Potential net sales milestones | Licensing And Collaboration Agreement                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Revenue Recognition, Milestone, Potential Achievements $ 325,000,000                           325,000,000    
Potential net sales milestones | Early Stage Collaborations                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Revenue Recognition, Milestone, Potential Achievements                             $ 252,000,000    
Minimum                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Term of Grant Program                             2 years    
Minimum | Collaboration And Discovery Agreements [Member]                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Collaborative Arrangements Research Period for Applying Discovery Technology                             3 years    
Maximum                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Term of Grant Program                             5 years    
Maximum | Collaboration And Discovery Agreements [Member]                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Collaborative Arrangements Research Period for Applying Discovery Technology                             4 years    
United States                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Net Product Sales                             $ 92,000,000    
Revenue                             93,694,000 $ 49,155,000  
Non-US                                  
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]                                  
Net Product Sales                             171,000,000    
Revenue                             $ 171,040,000 $ 145,237,000