Frataxin
The purpose of this research study is to determine a way to measure frataxin messenger RNA (mRNA) in fluids such as blood and cerebrospinal fluid (CSF) from patients with Friedreich's ataxia (FRDA). The gene mutation in FRDA leads to low levels of…
- Investigator
- Sub Subramony
- Status
- Accepting Candidates
- Ages
- 18 Years - 65 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
ARTHREX
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a…
- Investigator
- Sub Subramony
- Status
- Accepting Candidates
- Ages
- 18 Years - 64 Years
- Sexes
- All