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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

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

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