Spinal Muscular Atrophy

Spinal Muscular Atrophy (SMA) is a devastating genetic disorder affecting ~ 1:10,000 live births,  where the patient is incapable of naturally producing appropriate levels of survival motor neuron protein (SMN),  a key protein required for proper spinal cord and central nervous system development. Shift’s lead compound (E1v1.11) binds to a back-up gene (called SMN2) and converts the nominally non-function SMN2 protein into a full-length protein (similar to SMN1) within the patient’s spinal cord (and to a lower degree peripheral tissues).  In animal studies, this compound has significantly improved physical outcomes compared to currently available therapeutic treatments.

For more information about SMA and organizations actively pursuing treatment for SMA click on a logo below: