Consider appropriate patient types. Scarlette R., 17 years old. Student living with SMA. Scarlette hasn't received ITVISMA. Individuals shown were compensated for their time.

Oliver

History:
Age: 16 years old¹
Treatment duration: 4 years, 8 months (following 7 years of supportive care)
Current SMA treatment: not currently taking an SMN-targeted therapy. Tried risdiplam and nusinersen in the past few years, but did not continue due to perceived lack of efficacy¹

Milestones¹:
Sit without support: YES
Stand without assistance: YES
Walk without assistance: NO

Current status:
After a long period of clinical stability, Oliver feels his motor function is starting to decline. As he tackles all the changes and challenges of being a teen, spinal muscular atrophy (SMA) progression is simply not part of his plan.

Considerations:
Oliver values his independence and wants to keep up with friends at school and in extracurricular activities. He chose to stop SMA treatments when he felt they weren’t supporting his priorities. Now he is interested in treatment again and likes the idea of a one-time dose.

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.