Hereditary Inclusion Body Myopathy (HIBM)
2
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
2
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Genome & CompanyManNAc
Ultragenyx PharmaceuticalSialic Acid Extended Release
Clinical Trials (2)
Total enrollment: 52 patients across 2 trials
Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
Start: Sep 2012Est. completion: May 201326 patients
Phase 1Completed
Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
Start: Jul 2011Est. completion: May 201226 patients
Phase 1Completed
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
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