Cure SMA is extremely pleased to announce a generous $620,000 gift has been made to the organization. The donation was made anonymously in honor of William N. Kanehann. Billy had SMA and died in 2013 at the age of 23. We are grateful for this amazing donation in memory of his life.
This gift will be dedicated to supporting new translational SMA research with the goal of identifying new drug targets which will lead to practical new drugs and combination therapies for SMA.
Funding for this research will help discover new systems, pathways and processes that are affected in SMA. Approaches that work on these new areas could then be used in combination with the current treatment approaches being tested in clinical trials and now approved that work on increasing SMN levels. This important work will allow us to develop maximally effective treatments for all types, ages and stages of SMA.
As the SMA research landscape has developed and the drug pipeline has grown to include the first-ever FDA approved treatment, the needs for new translational SMA research have also developed. Cure SMA continues to invest in research by funding the areas of greatest need, and where we are best positioned to make a significant difference.
To learn more, or to make a contribution to Cure SMA’s research efforts visit www.curesma.org.