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Dr Lynch receives grants to perform clinical trials for Solid, Larimar, Biogen, and PTC, and has previously received grants from Neurocrine and Design.","order":2,"name":"Ethics","label":"Conflict of interest","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Not applicable.","order":3,"name":"Ethics","label":"Ethics approval","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Not applicable.","order":4,"name":"Ethics","label":"Consent","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"MPL and DRL are responsible for the conceptualization, writing, and editing of the text and figures of this article. 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