The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
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 […]
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the publication, “Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy” in the peer-reviewed journal Human Molecular Genetics. The publication details preclinical studies demonstrating that a highly specific […]
The research group of Prof. Dr. Brunhilde Wirth, Head of the Institute of Human Genetics at the University Hospital Cologne and Chair of our Scientific Advisory Board, succeeded to identify calcineurin-like EF-hand Protein 1 (CHP1) reduction as a novel protective modifier for SMA. The results of this study have been published in Brain, an international highly renowned journal. […]
SMA research receives vital funds from SMA Europe. Based on the recommendations received by its Scientific Advisory Board, SMA Europe awarded a total of €357,800 Euros to 3 cutting-edge SMA research projects through its 9th Call for Research Proposals. The Scientific Advisory Board reviewed and scored 21 very high level SMA research proposals. As only […]