Spinal Muscular Atrophy (SMA) is an inherited disorder that affects nerves and muscles, which causes muscles to become increasingly weak. The cause is a shortage of the protein SMN, or ‘survival of motor neuron’. This protein is crucial for the function of nerves that control muscle activity. Without this protein, the motor neuron cells shrink and eventually die. This leads to muscle weakness seen in SMA..
Experts in novel medicines
















