Spinal muscular atrophy (SMA) is a progressive neuromuscular disease characterised by weakness of the muscles in the shoulders, hips, thighs and upper back. It may also weaken the muscles used for breathing and swallowing.1
SMA results from mutations in the SMN1 gene on chromosome 5q.1 These mutations cause a shortage of a protein called survival motor neuron (SMN), which results in the loss of nerve cells in the spine.1
SMA is a clinical spectrum of disease with disease severity linked to fewer numbers of SMN2 gene copies and younger age of symptom onset.1
age 4½ years*Infantile-onset (Type I) SMA
treated with SPINRAZA®
~5 years as of 12/18
1. SPINRAZA® Summary of Product Characteristics.