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

 

Acronym

UX053-CL101

GSD3

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