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Fabry Disease – A Market Perspective On The Emerging Pipeline

Fabry disease is a rare hereditary lysosomal storage disorder that is caused by mutation in the GLA gene located on the X chromosome. The defect leads to the deficiency of an enzyme called alpha-galactosidase A, responsible for the breakdown of globotriaosylceramide (Gb3). The accumulation of the fatty substance le...

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Fabry Disease Treatment Landscape
Examining the Therapeutic Advances in Fabry Disease Treatment Landscape

Fabry Disease is an inherited lysosomal storage disease caused by a nonfunctional or partially functional enzyme called alpha galactosidase A (α-Gal A). Decreased activity of α-Gal A in lysosomes results in the accumulation of enzyme substrates (Gb3 and lyso-Gb3) which cause cellular damage in tissues throughout th...

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Alnylam’s Onpattro Receives First-Ever US FDA Approval for Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults US FDA approved Alnylam’s Onpattro (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneurop...

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