CTRI/2018/03/012267
Not yet recruiting
未知
Genzyme Rare disease registries (Pompe Disease Registry Protocol)
Sanofi Synthelabo India Private Limited0 sites0 target enrollmentTBD
Overview
- Phase
- 未知
- Intervention
- Not specified
- Conditions
- Health Condition 1: E740- Glycogen storage diseaseHealth Condition 2: null- Pompe Disease
- Sponsor
- Sanofi Synthelabo India Private Limited
- Status
- Not yet recruiting
- Last Updated
- 4 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\.All age group from new born to old age allowed.
- •2\. All patients with a confirmed diagnosis of Pompe disease who are willing and able to provide written informed consent and any additional authorization documents required by local law to send health information to the Registry are eligible for inclusion, regardless of whether they are receiving disease therapy including ERT (such as alglucosidase alfa) and irrespective of the commercial product with which they are being treated.
- •3\. A confirmed diagnosis is defined as documented acid α\-glucosidase (GAA) enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.
Exclusion Criteria
- •No Exclusion Criteria
Outcomes
Primary Outcomes
Not specified
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