Study SM202 This is a Phase II, Long term Ongoing 2019 randomised, double blind, safety, sham procedure tolerability, controlled study to assess the safety, tolerability, and efficacy pharmacokinetic, and data for efficacy in patients who patients with were not eligible to infantile and participate in Studies onset SMA CS3B or CS4. In light of assessed for up emergent data, Part 1 of to around 43 the study was terminated months. early and all subjects were Cardiac. rolled over into the open label Part 2 of the study. MDA US Prospective longitudinal Missing Planned Preliminary Neuromuscular registry in a research information: study synopsis Disease agreement with the safety profile in 1 month after Registry Muscular Dystrophy patients with low EC. Association. As of January or higher SMN2 2017, 28 participating copy number clinics across the US, with and/or different 205 unique patients disease severity diagnosed across the from the majority spectrum of SMA. Data of patients in the collection generally include nusinersen patient demographics, SMN clinical copy numbers, motor programme (for milestones, vital status, example, Type 0 surgical history, and Type IV hospitalisations, SMA); safety medications, mobility, profile of patients scoliosis, other > 18 years comorbidities, nutritional therapies, pulmonary function and devices, and cause of death. International Longitudinal natural history Missing Ongoing Updates to be SMA study with the 3 regional information: provided in Consortium centres that comprise the safety profile in PSURs natural ISMAC (SMA Reach UK, patients with low history study Italian SMA Network, and or higher SMN2 Dr. Richard Finkel at copy number Nemours Children’s Health and/or different System). Outputs expected disease severity to include baseline from the majority characteristics of treated of patients in the patients and longitudinal nusinersen data on nusinersen clinical treatment patterns, motor programme (for function, respiratory example Type 0 function, hospitalisations, and Type IV and comorbidities. SMA); safety
Study SM202 This is a Phase II, randomised, double blind, sham procedure controlled study to assess the safety, tolerability, pharmacokinetic, and efficacy in patients who were not eligible to participate in Studies CS3B or CS4. In light of emergent data, Part 1 of the study was terminated early and all subjects were rolled over into the open label Part 2 of the study. Long term safety, tolerability, and efficacy data for patients with infantile and onset SMA assessed for up to around 43 months. Cardiac. Ongoing 2019 MDA US Neuromuscular Disease Registry Prospective longitudinal registry in a research agreement with the Muscular Dystrophy Association. As of January 2017, 28 participating clinics across the US, with 205 unique patients diagnosed across the spectrum of SMA. Data collection generally include patient demographics, SMN copy numbers, motor milestones, vital status, surgical history, hospitalisations, medications, mobility, scoliosis, other comorbidities, nutritional therapies, pulmonary function and devices, and cause of death. 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 (for example, Type 0 and Type IV SMA); safety profile of patients > 18 years Planned Preliminary study synopsis 1 month after EC. International SMA Consortium natural history study Longitudinal natural history study with the 3 regional centres that comprise the ISMAC (SMA Reach UK, Italian SMA Network, and Dr. Richard Finkel at Nemours Children’s Health System). Outputs expected to include baseline characteristics of treated patients and longitudinal data on nusinersen treatment patterns, motor function, respiratory function, hospitalisations, and comorbidities. 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 (for example Type 0 and Type IV SMA); safety Ongoing Updates to be provided in PSURs