News | SMA Europe

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. SMA My Way: stories on topics that matter most

    August is SMA Awareness month and to kick this off, we would like to showcase a great new, community-focused initiative called “SMA My Way“, which brings together the lived experiences of people across the world who live SMA THEIR way! This initiative was born as a result of a need highlighted by people living with […]


  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. The website for SMA Europe’s 3rd scientific congress on SMA is live!

    We are delighted to announce that our congress website is now live. The 3rd edition of our congress will present a unique opportunity for scientists and clinicians in the field of SMA to present their latest research, to exchange ideas, to discuss potential collaborations and to network. This event will bring together not only the […]


  4. 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 […]