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
GRASP-01-003
This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
- Investigators
- Carla Zingariello, Sub Subramony
- Status
- Accepting Candidates
- Ages
- 12 Years - 50 Years
- Sexes
- All
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鈥
- Investigator
- Sub Subramony
- Status
- Accepting Candidates
- Ages
- 18 Years - 65 Years
- Sexes
- All