The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
Six dedicated young PhD students from institutions across the UK are organising an online SMA research conference on 10th & 11th September. Find out more about them, their work and their interests outside the laboratory! INES BOEHM Ines completed her BSc in Molecular Biology from the University of Salzburg and Johannes Keppler University Linz (Austria), […]
Save the date for the UK SMA Research Online Conference, which will take place on 10th & 11th September between 1 and 4.30pm GMT. A committee of UK-based PhD students have got together to organise this event with the aim of giving early career researchers (ECRs) an opportunity to present their work. There will be […]
SMA research receives vital funds from SMA Europe. Based on the recommendations received by its Scientific Advisory Board, SMA Europe awarded a total of 544,900 Euros to 4 cutting-edge SMA research projects through its 10th Call for Research Proposals. The Scientific Advisory Board reviewed and scored 19 very high level SMA research proposals. As only […]
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 […]
Biogen presented data at the American Academy of Neurology 2019 conference on a potential biomarker for SMA. Biomarkers provide insight on disease progression. Researchers have found that raised plasma levels of a protein called pNF-H indicate neurological damage and have potential to predict disease activity. The advantage of pNF-H is that it is present in […]