Mucopolysaccharidoses
0
Pipeline Programs
4
Companies
3
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
3 companies ranked by most advanced pipeline stage
BioMarin PharmaceuticalNOVATO, CA
1 programA Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative DiagnosisN/A1 trial
Active Trials
Innovation PharmaceuticalsMA - Wakefield
1 programLongitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VIN/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
Innovation PharmaceuticalsLongitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
Clinical Trials (3)
Total enrollment: 185 patients across 3 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
NCT01521429Innovation PharmaceuticalsLongitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
Start: Aug 2009Est. completion: Jul 201955 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
4 companies competing in this space
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