profile of patients > 18 years. Longitudinal natural history Missing Ongoing Updates to be studies in a research information: provided in agreement with the safety profile in PSURs. Alliance to expand patients with low current registries to include or higher SMN2 nusinersen treatment copy number information. The Global SMA and/or different Patient Registry consists of disease severity 26 national patient from the majority registries representing 29 of patients in the countries (20 countries in nusinersen genetically confirmed clinical patients across the spectrum programme Type of SMA. IV SMA); safety profile of patients Data are self-reported > 18 years and/or provided by healthcare professionals. More than 5000 SMA patients worldwide have been enrolled in associated registries. 11: Long term safety and efficacy studies short name and title Rationale and study Due Date(s) objectives/study design Study CS11 (SHINE) First patient This is an 17 open November label dosed extension 2015 study in An open label extension study subjects with SMA who for patients with spinal previously participated Last patient Q3 2017 muscular atrophy who dosed in investigational previously participated in studies of nusinersen. investigational studies of The primary purpose of Last patient Q3 2022 ISIS 396443 (nusinersen). this study is to gather completed
profile of patients > 18 years. Longitudinal natural history studies in a research agreement with the Alliance to expand current registries to include nusinersen treatment information. The Global SMA Patient Registry consists of 26 national patient registries representing 29 countries (20 countries in genetically confirmed patients across the spectrum of SMA. Data are self-reported and/or provided by healthcare professionals. More than 5000 SMA patients worldwide have been enrolled in associated registries. Missing information: safety profile in patients with low or higher SMN2 copy number and/or different disease severity from the majority of patients in the nusinersen clinical programme Type IV SMA); safety profile of patients > 18 years Ongoing Updates to be provided in PSURs. 11: Long term safety and efficacy studies short name and title Rationale and study objectives/study design Due Date(s) Study CS11 (SHINE) An open label extension study for patients with spinal muscular atrophy who previously participated in investigational studies of ISIS 396443 (nusinersen). This is an open label extension study in subjects with SMA who previously participated in investigational studies of nusinersen. The primary purpose of this study is to gather First patient dosed 17 November 2015 Last patient dosed Q3 2017 Last patient completed Q3 2022
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