Clinicians from the French network of neuromuscular disease specialists followed a cohort of 37 children treated with nusinersen (Spinraza) between the ages of 2 months and 3 years for three years.
- The motor functions of these patients, now aged between 3 and 6, improved.
- Motor development was greater in children with three copies of the SMN2 gene, who were able to stand and walk with assistance, than in those with two SMN2 copies, who were unable to do so.
- Respiratory, bulbar and orthopedic improvements were also greater in those with three SMN2 copies.
- In contrast, all patients with two copies and 64% of those with three copies developed scoliosis.
