[PDF][PDF] Augmenting the SMN protein to treat infantile spinal muscular atrophy
JK Kim, UR Monani - Neuron, 2018 - cell.com
JK Kim, UR Monani
Neuron, 2018•cell.comSpinal muscular atrophy (SMA) is a common and oft-fatal pediatric neuromuscular disorder
caused by insufficient SMN protein. Now, two clinical trials (Mendell et al., 2017; Finkel et al.,
2017) demonstrate that restoring the protein is therapeutic, offering new treatment options
and renewed hope to SMA patients.
caused by insufficient SMN protein. Now, two clinical trials (Mendell et al., 2017; Finkel et al.,
2017) demonstrate that restoring the protein is therapeutic, offering new treatment options
and renewed hope to SMA patients.
Spinal muscular atrophy (SMA) is a common and oft-fatal pediatric neuromuscular disorder caused by insufficient SMN protein. Now, two clinical trials (Mendell et al., 2017; Finkel et al., 2017) demonstrate that restoring the protein is therapeutic, offering new treatment options and renewed hope to SMA patients.
cell.com