The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Roche to start MANATEE, a global combination study in early 2022

    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 […]


  2. Novartis stops development of branaplam for SMA

    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 […]


  3. Apitegromab granted Fast Track Designation in the US for the treatment of SMA

    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 […]