220+
SMA Patients Successfully Recruited
8+
Completed SMA Studies
69%
Faster Enrollment Than Industry Average
97%
Patient Retention Rate in SMA Trials
Spinal Muscular Atrophy Specializations
Comprehensive recruitment strategies for all types and stages of spinal muscular atrophy.
SMA Type 1 (Severe)
Infantile-onset with most severe motor impairment
- Gene therapy trials
- Respiratory support studies
- Early intervention protocols
SMA Type 2 (Intermediate)
Childhood-onset with moderate motor function
- Functional improvement studies
- Mobility preservation
- Quality of life trials
SMA Type 3 (Mild)
Later-onset with ambulatory function
- Walking preservation
- Strength maintenance
- Long-term outcomes
SMA Type 4 (Adult-Onset)
Adult presentation with mild symptoms
- Progression monitoring
- Work capacity studies
- Family planning research
Pre-Symptomatic SMA
Genetically confirmed but not yet symptomatic
- Prevention studies
- Early treatment trials
- Natural history research
SMA with Respiratory Issues
Patients requiring ventilatory support
- Respiratory function studies
- Ventilation optimization
- Pulmonary care trials