Barry J Byrne, MD, PhD

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

18 Years - N/A

Sexes

All

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