MEPSEVII (vestronidase alfa-vjbk)
METABOLIC DISEASE ENZYME REPLACEMENT - MUCOPOLYSACCHARIDOSIS · MUCOPOLYSACCHARIDOSIS TYPE VII
ULTRAGENYX PHAR
Drug class
Mucopolysaccharidosis VII (MPS VII) Treatment Agents
Mucopolysaccharidosis VII (MPS VII) treatment agents represent a specialized category of enzyme replacement therapies specifically developed to manage mucopolysaccharidosis type VII, an ultra-rare lysosomal storage disorder also known as Sly syndrome. Read more about Mucopolysaccharidosis VII (MPS VII) Treatment Agents
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Drugs in Mucopolysaccharidosis VII (MPS VII) Treatment Agents
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Mucopolysaccharidosis VII (MPS VII) treatment agents represent a specialized category of enzyme replacement therapies specifically developed to manage mucopolysaccharidosis type VII, an ultra-rare lysosomal storage disorder also known as Sly syndrome.
Although commonly grouped under this indication-based formulary heading, the therapeutic class is primarily represented by vestronidase alfa, an exogenous recombinant human beta-glucuronidase.
These agents function by binding to mannose-6-phosphate receptors on target cells, which facilitates lysosomal uptake and restores catabolism of accumulated glycosaminoglycans within affected tissues.
Consequently, they are indicated to mitigate progressive multisystemic, non-neurological manifestations in pediatric and adult patients with confirmed beta-glucuronidase deficiency.
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molinahealthcare.com
Mucopolysaccharidosis VII (MPS VII) treatment agents represent a specialized category of enzyme replacement therapies specifically developed to manage mucopolysaccharidosis type VII, an ultra-rare lysosomal storage disorder also known as Sly syndrome Although commonly grouped under this indication-based formulary heading, the therapeutic class is primarily represented by vestronidase alfa, an exogenous recombinant human beta-glucuronidase Consequently, they are indicated to mitigate progressive ...
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nih.gov
Mucopolysaccharidosis VII (MPS VII) treatment agents represent a specialized category of enzyme replacement therapies specifically developed to manage mucopolysaccharidosis type VII, an ultra-rare lysosomal storage disorder also known as Sly syndrome Although commonly grouped under this indication-based formulary heading, the therapeutic class is primarily represented by vestronidase alfa, an exogenous recombinant human beta-glucuronidase
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drugbank.com
These agents function by binding to mannose-6-phosphate receptors on target cells, which facilitates lysosomal uptake and restores catabolism of accumulated glycosaminoglycans within affected tissues
-
fda.gov
These agents function by binding to mannose-6-phosphate receptors on target cells, which facilitates lysosomal uptake and restores catabolism of accumulated glycosaminoglycans within affected tissues
-
europa.eu
Consequently, they are indicated to mitigate progressive multisystemic, non-neurological manifestations in pediatric and adult patients with confirmed beta-glucuronidase deficiency
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