Barry J Byrne, MD, PhD

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe…

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

0 Years - 17 Years

Sexes

All

This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Patients will receive a single intravenous (IV) infusion…

Investigators

Renata Shih, Barry J Byrne

Status

Accepting Candidates

Ages

4 Years - 17 Years

Sexes

Male

This study is an international, multi-center, study of Pompe disease patients that are currently receiving enzyme-replacement therapy (ERT). The purpose of this study is to find out if the co-administration of investigational new drugs ATB200 and…

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

18 Years - 75 Years

Sexes

All