Spinal Muscular Atrophy (SMA) and Clinical Research


SMA is a rare genetic disease that affects the nerves in the spinal cord, causing severe muscle weakness, loss of movement, and difficulty breathing. It is an autosomal recessive genetic disorder, which means that generally both parents must carry a mutated SMN1 gene for their baby to inherit SMA. Because each parent typically has only one mutated SMN1 gene, the parents do not have SMA. They are considered “carriers.” SMA affects approximately 1 out of every 10,000 babies.

While there are now treatment options, there is still no cure for SMA, and unmet medical needs remain.

Researchers conducting the RESPOND Study are examining the drug nusinersen to determine its safety and efficacy for children who have previously taken onasemnogene abeparvovec.

Why is this clinical research study needed?

  • There is an important medical and scientific need to research the safety and efficacy of different treatment options for children with SMA who didnot respond optimally to onasemnogene abeparvovec.
  • Participation in clinical research studies provides an opportunity to assess the safety of treatment options.

Clinical studies are conducted to answer questions about the disease and investigational approaches, providing information that may help with the development of future medical treatments.

External SMA Resources

Check out Cure SMA or the Muscular Dystrophy Association for more information and support for people living with SMA and their caregivers.

These organizations and the content contained within these links are not affiliated or endorsed by Biogen.