Efficacy of enzyme replacement therapy with alpha-glucosidase in patient with late-onset Pompe disease
Not Applicable
- Conditions
- Pompe.Glycogen storage disease
- Registration Number
- IRCT2017111120981N2
- Lead Sponsor
- Research Deputy of Islamic Azad University, Tehran Medical Branch
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 2
Inclusion Criteria
Late-onset Pumpe disease
No received enzyme replacement therapy
Exclusion Criteria
Pregnancy
Unwillingness to participate in the study
Advanced liver disease and renal failure
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Respiratory function. Timepoint: Every 6 months. Method of measurement: spirometery.;Muscular force. Timepoint: evry 6 months. Method of measurement: physical exam.
- Secondary Outcome Measures
Name Time Method Muscular Enzymes. Timepoint: evry 6 months. Method of measurement: lab data.