The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
Based on our request for an update, Biogen has provided us with the following information about new results from their NURTURE study, a study of pre-symptomatic patients with SMA, recently presented at the virtual Cure SMA Research & Clinical Care Meeting, which took place from 10th to 12th June 2020. NURTURE is an ongoing, Phase […]
Roche presented new data on Part 1 of the SUNFISH trial, as well as on the JEWELFISH study, at the virtual Cure SMA Annual Conference, which took place between 8th and 12th June. SUNFISH SUNFISH is a large (231 participants) global two-part study in children and adults, aged between 2 and 25 years, with Type […]
Scholar Rock reported on the recent progress and upcoming milestones for the TOPAZ Phase 2 clinical trial of SRK-015 in patients with Type 2 or 3 SMA, in the light of the COVID-19 pandemic. SRK-015 is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. The drug is […]
As part of our ongoing partnership and following our request to receive important and timely information about the risdiplam clinical development programme, Roche shares with us an update on part 2 of the FIREFISH clinical trial. FIREFISH Part 2 is a pivotal, global study evaluating risdiplam in infants aged 1 – 7 months old with […]
Based on our request for an update, Biogen has provided us with the following information on its global clinical study DEVOTE. The study is designed to evaluate the safety, tolerability and potentially improved efficacy of nusinersen when administered at a higher dose than currently approved for the treatment of SMA. The Phase 2/3 randomised, controlled, […]