Muscle Response to Enzyme Replacement Therapy in Pompe Disease

Status

UNKNOWN

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT00713453

Enrollment

Registered

2008-07-11

Start date

2007-01-31

Completion date

2019-12-31

Last updated

2015-03-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pompe Disease

Keywords

Pompe disease, muscle, enzyme replacement therapy

Brief summary

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

Interventions

None listed

Study design

Observational model

CASE_ONLY

Eligibility

Sex/Gender

ALL

Healthy volunteers

Yes

Inclusion criteria

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

Exclusion criteria

Design outcomes

Primary

Measure	Time frame
all morphology changes improvement	6 months

Countries

Taiwan

Contacts

Primary ContactYin-Hsiu Chien, MD

chienyh@ntu.edu.tw886-2-23123456

Outcome results