Mucopolysaccharidosis Type 7

1
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
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Ultragenyx Pharmaceutical
2 programs
1
UX003Phase 1/21 trial
UX003N/A1 trial
Active Trials
NCT02097251No Longer Available
NCT01856218Completed3Est. Jul 2016

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Ultragenyx PharmaceuticalUX003

Clinical Trials (2)

Total enrollment: 3 patients across 2 trials

NCT01856218Ultragenyx PharmaceuticalUX003

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Start: Nov 2013Est. completion: Jul 20163 patients
Phase 1/2Completed
NCT02097251Ultragenyx PharmaceuticalUX003

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

N/ANo Longer Available

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