GNE Myopathy
Pipeline by Development Stage
Competitive Landscape
4 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (6)
Total enrollment: 273 patients across 6 trials
Multi-Center Study of ManNAc for GNE Myopathy
An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy
An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy
A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
First-in-human Study of UX016 in GNEM
A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases
Phase Legend
Key Insights
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.