
FDA Gives Guidance on Developing Treatments for Fabry Disease
The guidance discusses clinical trial design features that can support approval of treatments of Fabry disease.
FDA published
According to FDA, Fabry disease is “a rare, X-linked, slowly progressive, lysosomal storage disorder caused by pathogenic variants (disease-causing mutations) in the galactosidase alpha (GLA) gene resulting in absent or deficient activity of the lysosomal enzyme α-galactosidase A (α-Gal A). The α-Gal A enzyme breaks down glycosphingolipids within lysosomes. α-Gal A deficient activity leads to progressive intralysosomal accumulation of the undegraded substrate globotriaosylceramide (GL-3, also known as Gb3), a glycosphingolipid.” The disease affects both males and females despite being X-linked; although, males experience the most severe symptoms.
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