MDA’s legacy of progress has always started with families at the heart of all we do. For more than 70 years, we have been committed to saving and improving the lives of kids and adults living with muscular dystrophy and related life-threatening diseases. 

MDA is funding research that identifies, develops and tests strategies that restore sufficient levels of the needed SMN protein. Even though a great deal already is known about the SMA disease process, MDA-supported investigators in laboratories around the world continue to pursue basic research – the preclinical studies of underlying mechanisms and disease processes that lay the foundation for identifying promising therapy candidates.

Although a great deal of SMA research is aimed at increasing SMN levels, investigators don’t yet know whether doing so after the onset of the disease will rescue nerve cells. Therefore, some MDA-supported research is focused not on increasing SMN, but on improving muscle strength and function through other means. These non-SMN-based strategies hold promise for all forms of SMA, including X-linked SMA and SMA-LED.