Higher Dose of Alglucosidase Alpha for Pompe Disease

Taipei Veterans General Hospital

Status: Not yet enrolling

Conditions: Glycogen Storage Disease Type II

Treatments: Drug: Alglucosidase Alfa

Study type: Observational

Funder types: Other

Identifiers

NCT05017402

2021-02-019A

Details and patient eligibility

About

This study is aimed to investigate that whether the higher dose ERT improve safety and clinical outcomes of Pompe disease patients. Also, wish to develop a new therapeutic recommendation and hope that it could improve the long-term outcomes of Pompe diesease patients.

Enrollment: 36 estimated patients

Sex: All

Ages: Under 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Diagnosis of Pompe disease
2. Currently receiving ERT with regular clinic visits
3. Agree to sign informed consent

Exclusion criteria

1. Lost follow-up for clinical visits
2. Allergy to Myozyme
3. Refuse to sign informed consent