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Summary of significant accounting policies - Narrative (Details)
 1 Months Ended 3 Months Ended 12 Months Ended
Aug. 31, 2014
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 ($)
segment
Dec. 31, 2017
USD ($)
Dec. 31, 2016
USD ($)
Aug. 23, 2018
USD ($)
Jan. 01, 2018
USD ($)
Segment and geographic information                            
Number of operating segments | segment                   1        
Number of Reporting Units | segment                   1        
Minimum age of ambulatory patient 5 years                          
Allowance for doubtful accounts   $ 700,000       $ 800,000       $ 700,000 $ 800,000      
Deferred research and development advance payments   2,400,000       500,000       2,400,000 $ 500,000      
Impairment of long-lived assets                   $ 0        
Summary                            
Federal income tax provision at statutory rate                   21.00% 34.00% 34.00%    
Income tax benefit (expense)                   $ 29,000 $ (1,335,000) $ (569,000)    
Renewal Of Marketing Authorization, Period                   1 year        
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    
Accumulated deficit   (938,923,000)       $ (814,108,000)       (938,923,000) (814,108,000)      
Contract with Customer, Liability, Cumulative Catch-up Adjustment to Revenue, Change in Measure of Progress                   3,937,000        
Translarna                            
Summary                            
Net product revenue                   171,000,000 145,200,000 $ 81,400,000    
Emflaza                            
Summary                            
Net product revenue                   92,000,000 $ 28,800,000      
Agilis                            
Summary                            
Business Combination, Recognized Identifiable Assets Acquired and Liabilities Assumed, Deferred Tax Liabilities   $ 122,032,000               122,032,000     $ 115,200,000  
Difference between Revenue Guidance in Effect before and after Topic 606                            
Summary                            
Cumulative Effect Of New Accounting Principle In Period Of Adoption, Variable Consideration                   600,000        
Accumulated deficit                           $ 3,300,000
Minimum                            
Summary                            
Cumulative Effect Of New Accounting Principle In Period Of Adoption, Variable Consideration                   10        
Maximum                            
Summary                            
Cumulative Effect Of New Accounting Principle In Period Of Adoption, Variable Consideration                   $ 15