DID YOU KNOW? DTIC has over 3.5 million final reports on DoD funded research, development, test, and evaluation activities available to our registered users. Click HERE
to register or log in.
Identification of Novel Targets to Treat Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a debilitating neuromuscular disease that is one of the leading genetic causes of infant mortality. SMA is caused by insufficient levels of survival motor neuron (SMN) protein and is characterized by the loss of lower motor neurons in the anterior horn of the spinal cord and denervation-dependent muscle atrophy. Patients present with severe muscle weakness and fail to achieve normal motor milestones. Recently approved treatments for SMA, nusinersen and gene therapy, prolong lifespan, but only a small subset of patients have improvements in their motor function.
Approved For Public Release