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Revenue recognition - Narrative (Details)	1 Months Ended				3 Months Ended		12 Months Ended
	Oct. 31, 2017 USD ($)	Nov. 30, 2014 USD ($)	Jan. 31, 2014 USD ($)	Nov. 30, 2011 USD ($) compound	Mar. 31, 2018 USD ($) segment deliverable	Mar. 31, 2017 USD ($)	Dec. 31, 2017 USD ($) deliverable
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]
Number of operating segments | segment					1
Net product revenue					$ 55,981,000	$ 26,442,000
Contract with customer, liability							$ 11,891,000
Upfront cash payment					1,346,000
Licensing And Collaboration Agreement
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]
Upfront cash payment				$ 30,000,000
Revenue recognition, milestone method, revenue recognized	$ 20,000,000	$ 10,000,000	$ 7,500,000
Revenues					100,000	100,000
Licensing And Collaboration Agreement | Research And Development Event Milestones
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]
Revenue recognition, milestone, potential achievements					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 | Emflaza
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]
Net product revenue					19,200,000
Non-US | Translarna
Revenue, Initial Application Period Cumulative Effect Transition [Line Items]
Net product revenue					$ 36,800,000
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

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Represents the number of compounds included in preclinical development.

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Revenue Recognition, Milestone, Potential Achievements

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Amount of obligation to transfer good or service to customer for which consideration has been received or is receivable.

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Reference 1: http://www.xbrl.org/2003/role/presentationRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 45
-Paragraph 2
-URI http://asc.fasb.org/extlink&oid=82913815&loc=SL49130532-203044

Reference 2: http://www.xbrl.org/2003/role/presentationRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
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-Paragraph 8
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-URI http://asc.fasb.org/extlink&oid=82913847&loc=SL49130549-203045

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Number of operating segments. An operating segment is a component of an enterprise: (a) that engages in business activities from which it may earn revenues and incur expenses (including revenues and expenses relating to transactions with other components of the same enterprise), (b) whose operating results are regularly reviewed by the enterprise's chief operating decision maker to make decisions about resources to be allocated to the segment and assess its performance, and (c) for which discrete financial information is available. An operating segment may engage in business activities for which it has yet to earn revenues, for example, start-up operations may be operating segments before earning revenues.

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The amount of consideration recognized during the period for the milestone or milestones.

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Reference 1: http://www.xbrl.org/2003/role/presentationRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 605
-SubTopic 28
-Section 50
-Paragraph 2
-Subparagraph (e)
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Amount of revenue recognized from goods sold, services rendered, insurance premiums, or other activities that constitute an earning process. Includes, but is not limited to, investment and interest income before deduction of interest expense when recognized as a component of revenue, and sales and trading gain (loss).

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Reference 1: http://www.xbrl.org/2003/role/presentationRef
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-Name Accounting Standards Codification
-Topic 225
-SubTopic 10
-Section S99
-Paragraph 2
-Subparagraph (SX 210.5-03.1)
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