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Revenue recognition - Narrative (Details)	1 Months Ended	3 Months Ended		9 Months Ended
	Aug. 31, 2021 USD ($)	Sep. 30, 2021 USD ($) Distributor	Sep. 30, 2020 USD ($) Distributor	Sep. 30, 2021 USD ($) segment Distributor	Sep. 30, 2020 USD ($) Distributor	Dec. 31, 2020 USD ($)	Nov. 30, 2011 USD ($)
Revenue recognition
Number of operating segments | segment				1
Number of distributors | Distributor		2	2	2	2
Contract with customer, liability		$ 0		$ 0		$ 4,200,000
Contract with customer, asset		0		0		$ 0
Performance obligations satisfied in period			$ 2,000,000.0	4,000,000.0	$ 6,000,000.0
Deferred consideration payable		0		0
Remaining potential milestones that can be achieved		325,000,000.0		325,000,000.0
Revenue		$ 138,743,000	$ 118,404,000	$ 373,364,000	$ 261,902,000
Minimum
Revenue recognition
Percentage of net product sales threshold		10.00%	10.00%	10.00%	10.00%
Net product revenue
Revenue recognition
Revenue		$ 115,605,000	$ 82,708,000	$ 309,998,000	$ 226,143,000
Net product revenue | Non-US
Revenue recognition
Revenue		68,500,000	44,200,000	170,200,000	124,000,000.0
Emflaza | United States
Revenue recognition
Revenue		47,100,000	38,500,000	139,800,000	102,100,000
Translarna | Non-US
Revenue recognition
Revenue		67,200,000	43,400,000	166,300,000	122,700,000
Collaboration revenue
Revenue recognition
Revenue		10,011,000	35,000,000	30,018,000	35,063,000
Royalty revenue
Revenue recognition
Revenue		13,127,000	696,000	33,348,000	696,000
SMA License Agreement | Collaboration revenue
Revenue recognition
Revenue		10,000,000.0	35,000,000.0	30,000,000.0	35,100,000
SMA License Agreement | Royalty revenue
Revenue recognition
Revenue		13,100,000	700,000	33,300,000	700,000
SMA License Agreement | Roche And SMA Foundation | Collaboration revenue
Revenue recognition
Revenue		10,000,000.0	$ 35,000,000.0	30,000,000.0	$ 35,100,000
Research And Development Event Milestones | SMA License Agreement
Revenue recognition
Milestone payments received	$ 10,000,000.0
Research And Development Event Milestones | SMA License Agreement | Maximum
Revenue recognition
Remaining potential milestones that can be achieved							$ 135,000,000.0
Sales Milestones | SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved		$ 325,000,000.0		$ 325,000,000.0
Sales Milestones | SMA License Agreement | Maximum
Revenue recognition
Remaining potential milestones that can be achieved							$ 325,000,000.0

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