Spinal Muscular Atrophy: Patient-reported outcomes
Patient-reported outcomes after stem cell therapy for Spinal Muscular Atrophy, based on 18 patients in our follow-up registry.
Updated ·
Overall patient-reported outcomes
Physical condition
reported physical improvement
83%
N = 18
Quality of life
reported quality-of-life improvement
95%
N = 18
Treatment satisfaction
satisfied with the treatment outcome
89%
N = 18
Reported change vs. before treatment
Worse No change Small Moderate Significant
| Symptom | Reported change vs. before treatment | Reported improvement |
|---|---|---|
| Energy | | 100% |
| Limb muscle strength | | 94% |
| Hand control | | 93% |
| Movement in general | | 88% |
| Balance | | 88% |
| Trunk control | | 88% |
| Overall strength | | 88% |
| Trunk muscle strength | | 88% |
| Fine motor control | | 87% |
| Range of movement | | 81% |
| Swallowing | | 78% |
| Standing up | | 73% |
| Spasticity | | 60% |
| Walking | | 43% |
| Crawling | | 39% |
About this data
Patients self-assess each symptom on a 5-point scale (Worse / No improvement / Small / Moderate / Significant) at follow-up checkpoints after treatment, comparing to their pre-treatment baseline. "Reported improvement" combines the small, moderate and significant buckets. Data is updated daily from our internal patient registry. As with any medical treatment, past results do not guarantee future outcomes — improvements vary from patient to patient.
