Type III Glycogenosis

  • UX053-CL101: A Phase 1/2, First -in-Human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label), and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients with GSD III
  • GSD3: French observatory for patients with type III glycogenosis





Intervention UX053
Principal investigator Marion Masingue Karim Wahbi
Sponsor Ultragenix phamaceutical AIM
Study status In preparation In preparation
Recruitment status In preparation In preparation
Population Adult Adult