Barry J Byrne, MD, PhD

The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

2 Years - 3 Years

Sexes

Male

This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

N/A - 17 Years

Sexes

All

The purpose of this study is to learn more about the disease progression in patients with a TECPR2 mutation.

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

18 Months - 12 Years

Sexes

All