Barry J Byrne, MD, PhD

The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized…

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

4 Years - 7 Years

Sexes

Male

The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5).

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

6 Years - 13 Years

Sexes

All

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