Vertex Drug
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Investigator
- Sub Subramony
- Status
- Accepting Candidates
- Ages
- 18 Years - 64 Years
- Sexes
- All
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