85+
Pompe Patients Successfully Recruited
4+
Completed Pompe Studies
78%
Faster Enrollment Than Industry Average
98%
Patient Retention Rate in Pompe Trials
Pompe Disease Recruitment Specializations
Comprehensive recruitment strategies for all forms and stages of Pompe disease.
Infantile-Onset Pompe
Classic severe form with early cardiac and muscle involvement
- Enzyme replacement trials
- Cardiac function studies
- Respiratory support research
Late-Onset Pompe
Adult and juvenile forms with progressive muscle weakness
- Mobility preservation
- Respiratory function trials
- Exercise capacity studies
Treatment-Naive Pompe
Patients not yet receiving enzyme replacement therapy
- Natural history studies
- Treatment initiation trials
- Biomarker research
ERT-Experienced Pompe
Patients currently on enzyme replacement therapy
- Dose optimization
- Combination therapies
- Long-term efficacy studies
Pompe with Respiratory Issues
Patients with significant breathing difficulties
- Ventilatory support studies
- Pulmonary function trials
- Sleep disorder research
Pompe with Cardiac Issues
Patients with heart involvement and cardiomyopathy
- Cardiac protection studies
- Heart function monitoring
- Arrhythmia prevention