Scientists manage to optimize an available therapy, based on in vivo trials, to treat spinal muscular atrophy
Spinal muscular atrophy (SMA) is a rare neurodegenerative disease that affects motor neurons and is caused by mutations in both copies of the SMN1 gene. Although there are different types of SMA, which are determined by the severity of the disease and the time when symptoms appear, this disease is characterized by a progressive loss of muscle...