Spinal Muscular Atrophy: From Genes to Therapies
Understanding a Rare Neuromuscular Disease Spinal muscular atrophy (SMA) is a genetic disorder that causes muscle weakness and atrophy. It is characterized by the progressive loss of motor neurons, which are the nerve cells that control muscle movement. These neurons control voluntary muscle movements, including those involved in walking, swallowing, and breathing. Without sufficient SMN […]