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The purpose of this pilot project is to see if a supplemental form of dietary gangliosides can serve as a potential treatment for the rare metabolic condition called ganglioside GM3 synthase deficiency.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Supplementation of gangliosides | Experimental | Supplementation of dairy-derived concentrated gangliosides |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Supplementation of dairy-derived concentrated gangliosides. | Dietary Supplement |
|
| Measure | Description | Time Frame |
|---|---|---|
| Body length compared to normal pediatric growth curves. | Quarterly measures- change from baseline over 24 months. | |
| Change in scores of standardized developmental assessments (Vineland & Batelle) | Quarterly measures - change from baseline over 24 months | |
| Body Weight compared to normal pediatric growth curves | Quarterly measurements from baseline over 24 months | |
| Head circumference compared to normal pediatric growth curves. | Quarterly measures from baseline over 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Concentration of ganglioside GM3 in blood plasma | Six times per year over 24 months |
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Inclusion Criteria:
Exclusion Criteria:
-
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| Name | Affiliation | Role |
|---|---|---|
| Heng Wang, MD PhD | DDC Clinic | Principal Investigator |
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| ID | Term |
|---|---|
| C563799 | Amish Infantile Epilepsy Syndrome |
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