The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
Following our request for regular updates, Roche has shared an overview of their work in the community over the past year and a summary of progress in advancing research and increasing access to risdiplam in Europe and beyond. Clinical trial data updates Throughout 2021, Roche presented findings from the four ongoing clinical trials of risdiplam […]
Data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies aged 1-7 months old with symptomatic Type 1 SMA, has been published in the New England Journal of Medicine. The study met its primary endpoint with 29% of infants sitting without support for at least five […]
In response to our request, Roche has shared new interim data from two studies evaluating risdiplam in people with SMA, who have previously been treated with other SMA-targeting therapies (JEWELFISH) and pre-symptomatic babies (RAINBOWFISH). The findings were presented at the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting, which is taking place from […]
The Summary of Product Characteristics (SmPC) for Zolgensma has been updated. The SmPC is a document describing the properties and the officially approved conditions of use of a medicine. Summaries of product characteristics form the basis of information for healthcare professionals on how to use the medicine safely and effectively. Novartis Gene Therapies is committed […]
Following our request for regular updates, Roche is delighted to inform us that today the European Commission (EC) approved Evrysdi™ (risdiplam), the first and only at home treatment for SMA, with proven efficacy in adults, children and babies two months and older. Risdiplam was reviewed under the European Medicine Agency’s (EMA) accelerated assessment programme, intended […]