The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
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 […]
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 […]
Today is World Children’s Day and Jane Velkovski, 11-year-old disability advocate from North Macedonia, son of our member representative Denica Velkovska, joined UNICEF at a global summit at the United Nations’ Headquarters in New York where children and young people are leading a call to action to protect and promote child rights. The UNICEF summit […]
It was announced today that AveXis received FDA approval for Zolgensma®, the first gene therapy for paediatric patients with SMA. Zolgensma has been approved in the United States for the treatment of patients younger than 2 years of age who have been diagnosed with SMA and including those who were pre-symptomatic at diagnosis. This treatment […]
Over 250 rare disease stakeholders gathered in Brussels on 13th & 14th February to develop and endorse Eurordis’ new proposed framework to sustainably improve patient access to rare disease therapies. Mencía de Lemus, President of SMA Europe, delivered a clear message. Despite the speed with which treatments are being developed for SMA, many challenges still […]