BOB体育

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-na茂ve pediatric subjects with Pompe鈥�

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鈥�

Investigator

Barry J Byrne

Status

Accepting Candidates

Ages

4 Years - 17 Years

Sexes

Male