Overview

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

Status:
Completed
Trial end date:
2013-11-01
Target enrollment:
Participant gender:
Summary
GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA).
Phase:
Phase 2
Details
Lead Sponsor:
Ultragenyx Pharmaceutical Inc