Other Rare Disease Specializations
Comprehensive recruitment strategies for ultra-rare conditions and orphan diseases across all therapeutic areas.
Ultra-Rare Genetic Disorders
Conditions affecting fewer than 1 in 50,000 people
- Global patient identification
- Natural history studies
- Compassionate use programs
Metabolic Disorders
Rare inborn errors of metabolism and enzyme deficiencies
- Enzyme replacement trials
- Substrate reduction therapy
- Gene therapy studies
Connective Tissue Disorders
Rare collagen and connective tissue diseases
- Ehlers-Danlos studies
- Marfan syndrome trials
- Osteogenesis imperfecta research
Rare Skin Conditions
Dermatological conditions with limited treatment options
- Epidermolysis bullosa
- Ichthyosis studies
- Rare genodermatoses
Orphan Neurological Diseases
Rare neurological and neurodegenerative conditions
- Ataxia studies
- Rare epilepsies
- Mitochondrial disorders
Rare Pediatric Conditions
Ultra-rare diseases affecting children specifically
- Developmental disorders
- Rare syndromes
- Pediatric cancer subtypes
Custom Recruitment Approaches
Tailored strategies for each unique rare disease condition and study requirement.
Global Patient Networks
Access to international rare disease registries and patient organizations worldwide.
Compassionate Use Programs
Expertise in expanded access and named patient programs for urgent cases.
Natural History Studies
Long-term follow-up and disease progression studies for research purposes.
Genetic Screening Programs
Family-based recruitment and cascade screening for hereditary conditions.