The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
We are delighted to have welcomed three new organisations into our fold: MDA Hellas (Greece) MDA Hellas is a muscular disease association founded in 2000, which focuses on improving the lives of individuals with neuromuscular disorders: not only on forging a lasting working relationship with government agencies and on enlisting the help of private institutions […]
SMA Europe is delighted to announce that Nicole Gusset has been appointed as President. This appointment is effective immediately and follows Mencía de Lemus’ decision to step down as president of SMA Europe. We are fortunate to have someone of Nicole Gusset’s calibre step up to lead SMA Europe. Nicole has a scientific background, working […]
AveXis, which has been part of Novartis since 2018, is becoming Novartis Gene Therapies, EU Ltd. (Novartis Gene Therapies) a new unit dedicated to developing innovative AAV-based gene therapies. While the name of the organisation changes, the commitment to patients and their families remains steadfast. Novartis Gene Therapies will continue to focus on the research, […]
On 19th May, the European Commission issued a conditional marketing authorisation for the breakthrough gene therapy drug onasemnogene abeparvovec (Zolgensma®) for treating spinal muscular atrophy. This is the second drug developed to treat this severe neuromuscular disorder to receive approval in the European Union. Onasemnogene abeparvovec, marketed as Zolgensma®, is authorised for use in treating […]
The European Reference Network for Neuromuscular diseases (Euro-NMD) wants to increase its understanding of the effects COVID-19 has on patients with existing neuromuscular conditions. With this in mind, they have launched a survey for neuromuscular patients and clinicians. They would be grateful if you could take a few moments to complete this online form to […]