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Revenue recognition - Collaboration and Royalty Revenue (Details) $ in Thousands	1 Months Ended	3 Months Ended			6 Months Ended	9 Months Ended
	Jan. 31, 2025 USD ($)	Sep. 30, 2025 USD ($)	Sep. 30, 2024 USD ($)	Jun. 30, 2024 USD ($)	Jun. 30, 2024 USD ($)	Sep. 30, 2025 USD ($) product	Sep. 30, 2024 USD ($)	Dec. 31, 2024 USD ($)	Nov. 30, 2011 USD ($)
Revenue recognition
Number of approved products \| product						2
Remaining potential milestones that can be achieved								$ 150,000
Revenue		$ 211,007	$ 196,786			$ 1,565,978	$ 593,608
Deferred revenue		1,691				1,691		$ 5,505
Collaboration and license revenue
Revenue recognition
Revenue		9,258				998,430
Royalty revenue
Revenue recognition
Revenue		70,793	61,365			$ 164,837	145,702
SMA License Agreement
Revenue recognition
Number of approved products \| product						1
SMA License Agreement \| Collaboration and license revenue
Revenue recognition
Revenue				$ 0	$ 0
SMA License Agreement \| Royalty revenue
Revenue recognition
Revenue		70,800	$ 61,400			$ 164,800	$ 145,700
License and Collaboration Agreement
Revenue recognition
Refund of revenue related to prior collaboration arrangements						3,500
License and Collaboration Agreement \| Novartis
Revenue recognition
Cash proceeds from upfront payment on collaboration agreement	$ 1,000,000
Percentage share of the income (loss) generated on U.S. sales	40.00%
Deferred revenue		0				0
License and Collaboration Agreement \| Novartis \| Collaboration and license revenue
Revenue recognition
Revenue		9,300				1,000,000
Research And Development Event Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved		0				0
Sales Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved		$ 150,000				$ 150,000
Maximum \| License and Collaboration Agreement \| Novartis
Revenue recognition
Potential future proceeds contingent on achievement of developmental, regulatory and sales milestones and tiered royalties	$ 1,900,000
Maximum \| Research And Development Event Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved									$ 135,000
Maximum \| Sales Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved									$ 325,000