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For Seniors
For adults
Recruiting in 10s
Interventional Study
For Males
Gastroenterology
Recruiting

A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

The GLA enzyme is ubiquitously expressed throughout the body and is responsible for the breakdown of glycosphingolipids, deficiency of which results in the accumulation of specific glycosphingolipids that are associated with the pathophysiology of the disease.

deficiency

fabry's disease

glycosphingolipids

gene therapy

breakdown

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19 Feb, 2024

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