Sanfilippo Syndrome Type C
0
Pipeline Programs
1
Companies
2
Clinical Trials
1 recruiting
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Phoenix NestNY - Brooklyn
2 programsNatural History Study of Participants With Sanfilippo Syndrome Type IIICN/A1 trial
Natural History to Assess Disease in Patients With MPS IIICN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2024
2025
2026
2027
2028
2029
Phoenix NestNatural History to Assess Disease in Patients With MPS IIIC
Phoenix NestNatural History Study of Participants With Sanfilippo Syndrome Type IIIC
Clinical Trials (2)
Total enrollment: 65 patients across 2 trials
Natural History to Assess Disease in Patients With MPS IIIC
Start: Nov 2026Est. completion: Feb 202935 patients
N/ANot Yet Recruiting
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
Start: Nov 2024Est. completion: Dec 202830 patients
N/ARecruiting
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 actively recruiting trials targeting 65 patients
1 companies competing in this space
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.