Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
| Status: | Completed |
|---|---|
| Conditions: | Metabolic |
| Therapuetic Areas: | Pharmacology / Toxicology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 11/8/2014 |
| Start Date: | June 2012 |
| End Date: | June 2014 |
| Contact: | Elizabeth (Beth) E Woolley, MS |
| Email: | elizabeth.e.woolley@emory.edu |
| Phone: | 404-778-8616 |
Discovering New Biomarkers for Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA (MPSIVA).
The purpose of this study is to find out more about some of the unusual chemicals (called
oligosaccharides) that can occur naturally as a result of processes in the body.
Researchers want to look at how these chemicals change with time and how they change between
different patients with MPSIVA. These unusual chemicals were recently discovered in the
urine from patients with MPSIVA. The investigators would like to study these chemicals
before a specific enzyme replacement therapy is used. If the investigators understand how
these chemicals change, the investigators may be able to use them to monitor this condition
in the near future as well as help doctors know whether certain therapies work well in their
patients.
oligosaccharides) that can occur naturally as a result of processes in the body.
Researchers want to look at how these chemicals change with time and how they change between
different patients with MPSIVA. These unusual chemicals were recently discovered in the
urine from patients with MPSIVA. The investigators would like to study these chemicals
before a specific enzyme replacement therapy is used. If the investigators understand how
these chemicals change, the investigators may be able to use them to monitor this condition
in the near future as well as help doctors know whether certain therapies work well in their
patients.
This is not a clinical trial and there is no outcome measurement. The biomarkers in this
study are oligosaccharides that secreted in the urine from patients with MPSIVa. In this
study, we will try to define the variability of these oligosaccharides in the same patient
at different time points, including different month of a year and different collections
during the day as well as variations of these oligosaccharide in different affected
patients.
study are oligosaccharides that secreted in the urine from patients with MPSIVa. In this
study, we will try to define the variability of these oligosaccharides in the same patient
at different time points, including different month of a year and different collections
during the day as well as variations of these oligosaccharide in different affected
patients.
Inclusion Criteria:
- Diagnosis of Mucopolysaccharidosis IVA
Exclusion Criteria:
- Patients receiving enzyme replacement therapy.
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