The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
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 […]
On 27th March, the European Medicines Agency recommended a conditional approval of the breakthrough gene therapy drug onasemnogene abeparvovec (Zolgensma®) for treating spinal muscular atrophy. SMA Europe is thrilled that a second therapy that addresses the causes of this life-limiting disease has moved closer to patients. It is a culmination of efforts that have been […]
Dear community, This strain of coronavirus is new, which means the knowledge on how it will affect people living with SMA is still limited. However, this is a rapidly changing field so advice will be updated as more is learned. We have built a repository of information, where this information will be uploaded. Leading experts […]