cebsulfase alfa

cebsulfase alfa (formerly known as SHP611) is a recombinant human arylsulfatase A (rhASA), developed as an enzyme replacement therapy for metachromatic leukodystrophy (MLD), a rare lysosomal storage disorder caused by deficient activity of the enzyme arylsulfatase A. The drug is administered intrathecally and aims to break down accumulated sulfatides in the central nervous system that result from the enzymatic deficiency in MLD. Clinical studies have shown that intrathecal administration of cebsulfase alfa can reduce cerebrospinal fluid sulfatide levels and may slow neurological decline in patients with late infantile MLD. The drug was originally developed by Shire Human Genetic Therapies and is now being further developed by Takeda[1][2][3][5][6].

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Metazym

Other names

recombinant human arylsulfatase ArhASA