The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
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 […]
In response to our request, Biogen has provided us with information and addressed our questions and concerns regarding the upcoming SPINRAZA (nusinersen) EU label (Prescribing information and Package Leaflet) update that will include information on the potential risk of hydrocephalus. All medicines are evaluated for the benefit they provide to patients as well as the […]
In April this year, a new 2-year strategy was set and a new working structure established. Our new structure Elections Presidency: Mencía de Lemus from our Spanish organisation, FundAME vice-Presidency: Nicole Gusset from our Swiss organisation, SMA Schweiz Treasury: Inge Schwersenz from our German organisation, DGM Executive Committee As SMA Europe continues to grow, it […]