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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). Patients will be treated sequentially in 2…

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

6 Years - 13 Years

Sexes

All

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