April 6, 2018
Spinal muscular atrophy is caused when an individual has two missing or faulty SMN1 genes. But what is the SMN1 gene, and what does it do?
About the SMN1 Gene
SMN1 stands for survival motor gene 1. This gene is responsible for...
March 21, 2018
Cure SMA has awarded a $150,000 research grant to Charlotte Sumner, MD, at Johns Hopkins University, for her project, "Testing the potential of SMN-AS1 as a therapeutic target in SMA.”
Dr. Sumner and her team have recently identified a long noncoding RNA (lncRNA) that represses...
March 20, 2018
In 2016, the spinal muscular atrophy community celebrated the approval of the first-ever treatment that targets the underlying genetic cause of SMA. This was a milestone more than 100 years in the making, made possible by dedicated researchers, and the community that supported their...