The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
In response to our request, Roche is pleased to inform us about the initiation of MANATEE, a new global Phase 2/3 clinical study to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody targeting muscle growth in combination with risdiplam in SMA. Why a combination study? This study draws on ten years […]
Biogen plans to initiate a new clinical trial called ASCEND, which will evaluate whether treatment with a higher dose of nusinersen has the potential to improve clinical outcomes and address unmet medical needs in people with later-onset SMA who were previously treated with risdiplam. Study overview The ASCEND study will assess if nusinersen at an […]
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 […]
Novartis has made the decision to discontinue development of branaplam, an investigational oral, once-weekly RNA splicing modulator, for the treatment of SMA. This was a difficult decision that was made as the result of rapid advancements in the SMA treatment landscape in recent years. Knowing the progressive nature of this devastating, genetic disease, maintaining continuity […]
Scholar Rock has received Fast Track Designation in the US from the FDA for apitegromab for the treatment of SMA. Apitegromab (also known as SRK-015) is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA. Fast Track status is granted to drugs that show considerable potential […]