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
Exclusion Criteria
- individuals with acid alpha glucosidase deficiency presenting symptoms
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method all muscle function 10 years
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What molecular mechanisms underlie disease progression in late-onset Pompe disease as observed in NCT00713245?
How does enzyme replacement therapy compare to natural disease progression in late-onset Pompe disease patients?
What biomarkers correlate with clinical outcomes in NCT00713245 late-onset Pompe disease cohort?
What are the long-term adverse events associated with Pompe disease management strategies in observational studies?
How do GAA enzyme activity levels and lysosomal function relate to prognosis in late-onset Pompe disease research?
Trial Locations
- Locations (1)
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
National Taiwan University Hospital🇨🇳Taipei, TaiwanYin-Hsiu Chien, MDSub InvestigatorWhu-Liang Hwu, MD, PhDPrincipal Investigator