DYNE-201 ACHIEVE
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).
The study consists of 4 periods: A Screening Period (up to…
- Investigator
- Sub Subramony
- Status
- Accepting Candidates
- Ages
- 18 Years - 65 Years
- Sexes
- All
Biogen 296FA301 BRAVE
In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older. However, it is not yet available for…
- Investigator
- Sub Subramony
- Status
- Accepting Candidates
- Ages
- 2 Years - 15 Years
- Sexes
- All
BrAAVe
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
The purpose of this study…
- Investigator
- Sub Subramony
- Status
- Accepting Candidates
- Ages
- 10 Years - 55 Years
- Sexes
- All