Current status:
It was considered a triumph when Sandra began grade school, but she’s experiencing increasing difficulty moving between rooms at her school. She often stays behind and does not participate in activities. 

Considerations:
Sandra’s parents recognize she’s reaching the age when motor symptoms tend to worsen without spinal muscular atrophy (SMA) treatment. Living in a rural area with limited access to neurology specialists, financial constraints, and insurance barriers has meant Sandra has only received supportive care to date. As Sandra’s mobility challenges increase, her parents are seeking a therapy that has potential to improve motor function as she grows, so as not to disrupt her schooling. Minimizing dosing requirements as part of her long-term SMA management plan may also support their goals as they plan for her future.

This hypothetical patient profile is based on patient experiences seen in clinical practice. This patient profile should not be used as a substitute for independent medical judgment. Models are for illustrative purposes and not actual patients. Individual results may vary. 

SMA, spinal muscular atrophy; SMN, survival motor neuron gene.