spinal muscular atrophy
Spinal muscular atrophy (SMA) is a genetic disorder that affects the motor neurons in the spinal cord. This leads to muscle weakness and atrophy, primarily impacting movement and coordination. SMA is caused by a deficiency of a protein called survival motor neuron (SMN), which is essential for the health of motor neurons.
There are several types of SMA, classified by the age of onset and severity. The most common form, SMA type 1, appears in infants and can significantly affect their ability to sit or crawl. Advances in treatment, such as gene therapy, have improved outcomes for many individuals with SMA.