Mucopolysaccharidosis IV Type A
0
Pipeline Programs
1
Companies
1
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
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
BioMarin PharmaceuticalVimizim®
Clinical Trials (1)
Total enrollment: 418 patients across 1 trials
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Start: Sep 2014Est. completion: Feb 2024418 patients
N/ACompleted
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