Mucopolysaccharidoses
0
Pipeline Programs
3
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
2 companies ranked by most advanced pipeline stage
BioMarin PharmaceuticalNOVATO, CA
1 programA Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative DiagnosisN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Ultragenyx PharmaceuticalCarotid ultrasonography
BioMarin PharmaceuticalA Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis
Clinical Trials (2)
Total enrollment: 130 patients across 2 trials
Cardiovascular Structure and Function in the Mucopolysaccharidoses
Start: Oct 2021Est. completion: Jun 202630 patients
N/AActive Not Recruiting
NCT03017677BioMarin PharmaceuticalA Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis
A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis
Start: Jan 2018Est. completion: Dec 2020100 patients
N/AUnknown
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
3 companies competing in this space