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A Long Term Follow up Study in Late-onset Pompe Disease

Conditions
Pompe Disease
Registration Number
NCT00713245
Lead Sponsor
National Taiwan University Hospital
Brief Summary

This study is to observe the progression of disease in late-onset Pompe disease

Detailed Description

Not available

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
70
Inclusion Criteria
  • individuals with acid alpha glucosidase deficiency without obvious symptoms
  • parents signed inform consent
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Exclusion Criteria
  • individuals with acid alpha glucosidase deficiency presenting symptoms
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Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
all muscle function10 years
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

National Taiwan University Hospital

🇨🇳

Taipei, Taiwan

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