Researchers seek biomarker to assess spinal muscular atrophy treatment

Spinal muscular atrophy (SMA) is the leading genetic cause of death in infants. As promising new therapies such as those directly targeting survivor motor neuron (SMN) are entering clinical trials for infants, children, and adults with SMA, researchers are searching for biomarkers in blood that can monitor their effectiveness….

Spinal muscular atrophy (SMA) is the leading genetic cause of death in infants. As promising new therapies such as those directly targeting survivor motor neuron (SMN) are entering clinical trials for infants, children, and adults with SMA, researchers are searching for biomarkers in blood that can monitor their effectiveness….