Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease. In its most common form, Type I SMA (~60% of cases), symptoms begin in infancy. Affected children never sit up, and they rapidly develop difficulty swallowing and breathing, eventually requiring feeding tubes and mechanical ventilation. Without treatment, children typically die by age 2

Treatments of Interest:

  • onasemnogene abeparvovec (Zolgensma®, Novartis AG/AveXis)
  • nusinersen (Spinraza®, Biogen)

For questions or additional information, please contact info@icer.org

View the Key Stakeholder List.

Final Documents

Below you will find the final documents from the assessment review process: