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Sly Syndrome

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

Ultragenyx Pharmaceutical
2 programs
UX003PHASE_21 trial
UX003PHASE_31 trial
Active Trials
NCT02418455Completed8Est. Mar 2019
NCT02432144Completed12Est. Jan 2019

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Ultragenyx PharmaceuticalUX003
Ultragenyx PharmaceuticalUX003

Clinical Trials (2)

Total enrollment: 20 patients across 2 trials

NCT02432144Ultragenyx PharmaceuticalUX003

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Start: Nov 2015Est. completion: Jan 201912 patients
Phase 3Completed
NCT02418455Ultragenyx PharmaceuticalUX003

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

Start: Jul 2015Est. completion: Mar 20198 patients
Phase 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.