The Spring 2024 issue of Compass is now available online.
This year, we are proud to announce six new research grants, totaling $750,000, to further our mission to drive breakthroughs in the treatment of spinal muscular atrophy (SMA). Basic research is the first step in developing new treatments and ultimately, a cure for spinal muscular atrophy (SMA).
These new projects investigate the biology and cause of SMA to identify the most effective strategies for drug discovery. Cure SMA has a rich history of supporting research aimed at improving quality of life, expanding treatment options, and advancing the understanding of SMA.
In this issue of Compass, you will meet our new recipients, learn about the important research they are conducting, and see how these awards may help advance treatment for people living with SMA.
There are now three treatments approved by the U.S. Food and Drug Administration (FDA) that target the underlying genetics of SMA—with several others in various phases of clinical trials. While the advent of these treatments for SMA have given families and individuals living with SMA hope for improved outcomes and longer life expectancies, we’re on the verge of further breakthroughs that will continue to change the course of SMA for everyone affected.
In the end, it will likely take a combination of therapeutic approaches—tailored to everyone’s age, stage, and type of SMA—to achieve the greatest possible effect for all patients with SMA. Basic research is our continued investment in further drug development for SMA. Without it, the SMA drug pipeline would not continue to grow and diversify.
The awarded researchers highlighted in Compass seek to explore novel targets for potential combination therapies and understand the pathology of SMA more fully. We thank these—and all researchers—for their ongoing commitment to the treatment of all people with SMA and to the SMA Community.
Thank You
Special thanks to the Concepcion Family, Nunemaker Family, Weisman Family, Luke 18:1 Foundation and Dhont Foundation for their generosity to Cure SMA in our quest to invest in basic research that will ultimately drive the next generation of SMA treatments.