HARBOR
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
- Investigator
- Sub Subramony
- Status
- Accepting Candidates
- Ages
- 16 Years - 65 Years
- Sexes
- All
ATA-200 gene therapy trial in patients with LGMDR5
The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5). Patients will be treated sequentially in 2…
- Investigator
- Barry J Byrne
- Status
- Accepting Candidates
- Ages
- 6 Years - 13 Years
- Sexes
- All
Fortitude OLE
A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
- Investigator
- Sub Subramony
- Status
- Accepting Candidates
- Ages
- 16 Years - 70 Years
- Sexes
- All