Spinal muscular atrophy (SMA) is a recessive neuromuscular disease that is caused by loss-of-function mutations in the survival motor neuron 1 (SMN1) gene and is one of the most common inheritable causes of infant death. In a recent study, researchers investigated baseline and drug-induced SMN levels in disease-relevant human tissue, find out what they learned.
For research use only. Not for use in diagnostic procedures.
Establishing a baseline and drug-induced SMN expression profile in SMA disease-relevant human tissues in expedited autopsies