News

Cure SMA has awarded a $150,000 research grant to Brunhilde Wirth, PhD, at Institute of Cologne, Germany, for her project, "Study of combinatorial therapy based on SPINRAZA together with a novel protective genetic modifier.”

Based on so-called SMA discordant families, in whom...

Scholar Rock today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug...

Cure SMA is thrilled to welcome Biogen back as a National Premier Partner for 2018.

As a part of their partnership, Biogen will be sponsoring all walk-n-rolls and galas across the US for the entirety of 2018, as well as the

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...

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...