Sanfilippo Syndrome Type A (MPS IIIA)
0
Pipeline Programs
1
Companies
2
Clinical Trials
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
Swedish Orphan BiovitrumMA - Waltham
2 programsSOBI003PHASE_1_21 trial
SOBI003PHASE_1_21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Swedish Orphan BiovitrumSOBI003
Swedish Orphan BiovitrumSOBI003
Clinical Trials (2)
Total enrollment: 12 patients across 2 trials
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Start: Jan 2019Est. completion: May 20216 patients
Phase 1/2Completed
A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
Start: Jun 2018Est. completion: Oct 20196 patients
Phase 1/2Completed
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 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.