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This document provides a summary of the 12-month results of Parts 1 and 2 of the FIREFISH study. The study started in December 2016 and met its key endpoints in November 2019, when the last baby to take part had completed 12 months of treatment with risdiplam, the drug being investigated in this study. The study will continue up to 5 years after the last baby has enrolled in the study.

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This document provides a summary of the 12-month results of Parts 1 and 2 of the SUNFISH study. The study started in October 2016 and met its key endpoints in September 2019, when the last person to take part had completed 12 months of treatment with risdiplam, the drug being investigated in this study. The study will continue up to 5 years after the last patient has enrolled in the study. This document has been written for members of the public, as well as the patients and families participating in the study.

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