Overview

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

Status:
Completed
Trial end date:
2021-01-31
Target enrollment:
Participant gender:
Summary
This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment).
Phase:
Phase 3
Details
Lead Sponsor:
PTC Therapeutics
Treatments:
Deflazacort