Muscle Response to Enzyme Replacement Therapy in Pompe Disease

National Taiwan University

Status: Unknown

Conditions: Pompe Disease

Study type: Observational

Funder types: Other

Identifiers

NCT00713453

200704066R

Details and patient eligibility

About

This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy

Enrollment: 16 estimated patients

Sex: All

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• patients with Pompe disease, prepare or on enzyme replacement therapy
• signed informed consent

Exclusion criteria