Health Condition 1: null- Fabry Disease Health Condition 2: E752- Other sphingolipidosis
Conditions
Interventions
None listed
Sponsors
Sanofi Synthelabo India Private Limited
Eligibility
Inclusion criteria
Inclusion criteria: 1. all age group from new born to old age allowed. 2. All patients with a confirmed diagnosis of Fabry 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 enzyme replacement therapy (ERT) (such as agalsidase beta) and irrespective of the commercial product with which they are being treated. 3. A confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte αGAL enzyme activity and/or mutation(s) in the gene coding for αGAL
Exclusion criteria
Exclusion criteria: No exclusion criteria, its a disease registry
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1) To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; 2) To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; 3) To characterize and describe the Fabry population as a whole; and 4) To evaluate the long-term safety and effectiveness of Fabrazyme® (agalsidase beta).Timepoint: 15 years | — |
Secondary
| Measure | Time frame |
|---|---|
| No Secondary OutcomeTimepoint: NA | — |
Countries
Argentina, Australia, Belgium, Brazil, Canada, China, Denmark, France, Germany, Greece, India, Ireland, Israel, Italy, Japan, Mexico, Netherlands, Norway, Peru, Republic of Korea, Russian Federation, Singapore, Spain, Taiwan, Turkey, United Kingdom, United States of America
Contacts
Public ContactMs Salini James
Sanofi Genzyme India
Outcome results
None listed