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Description of Business (Details)		1 Months Ended	3 Months Ended		12 Months Ended
Description of Business (Details)	Jul. 18, 2017 USD ($) shares	Sep. 30, 2018 USD ($) program	Sep. 30, 2018 USD ($)	Mar. 31, 2018 USD ($) shares	Dec. 31, 2018 USD ($) medicine program	Dec. 31, 2017 USD ($)	Dec. 31, 2016 USD ($)	Oct. 08, 2018 program
Corporate Information, Status of Operations and Management Plans
Number of approved medicines for Farby disease \| medicine					1
Number of gene therapy programs \| program					14			4
Number of gene therapy programs for Batten disease \| program					2
Number of gene therapy programs for neurologic LSDs developed \| program		10
Number of common stock shares issued from underwriting agreement \| shares	21,122,449			20,239,839
Net proceeds from the issuance of common stock after deducting underwriting discounts and commissions	$ 243,000,000			$ 294,600,000	$ 294,584,000	$ 243,037,000	$ 97,068,000
Accumulated deficit					$ (1,412,222,000)	$ (1,063,610,000)
Line of Credit
Corporate Information, Status of Operations and Management Plans
Maximum borrowing capacity		$ 150,000,000.0	$ 150,000,000.0
Debt instrument term		5 years	5 years
Proceeds from issuance of debt		$ 146,600,000
Line of Credit \| LIBOR
Corporate Information, Status of Operations and Management Plans
Basis spread on variable rate		7.50%	7.50%
Celenex
Corporate Information, Status of Operations and Management Plans
Research and development expense		$ 100,000,000