Barry J Byrne, MD, PhD

This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2…

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

4 Years - 13 Years

Sexes

Male

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