VIMIZIM (elosulfase alfa)
METABOLIC DISEASE ENZYME REPLACEMENT - MUCOPOLYSACCHARIDOSIS · MUCOPOLYSACCHARIDOSIS TYPE IV-A
BIOMARIN PHARMA
Drug class
Mucopolysaccharidosis IVA (MPS IVA) Treatment Agents
Mucopolysaccharidosis IVA (MPS IVA) treatment agents represent a therapeutic category composed of disease-specific therapies, primarily recombinant enzyme replacement therapy. Read more about Mucopolysaccharidosis IVA (MPS IVA) Treatment Agents
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Drugs in Mucopolysaccharidosis IVA (MPS IVA) Treatment Agents
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Mucopolysaccharidosis IVA (MPS IVA) treatment agents represent a therapeutic category composed of disease-specific therapies, primarily recombinant enzyme replacement therapy.
While not a standard formal FDA pharmacological class designation, this disease-directed group functions by supplying exogenous human N-acetylgalactosamine-6-sulfate sulfatase, represented by elosulfase alfa.
This mechanism facilitates the catabolism and cellular clearance of accumulated glycosaminoglycans, specifically keratan sulfate and chondroitin-6-sulfate, inside lysosomes.
Consequently, these agents are utilized to manage mucopolysaccharidosis type IVA, also known as Morquio A syndrome, helping to improve endurance and reduce systemic storage pathology.
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nih.gov
Mucopolysaccharidosis IVA (MPS IVA) treatment agents represent a therapeutic category composed of disease-specific therapies, primarily recombinant enzyme replacement therapy While not a standard formal FDA pharmacological class designation, this disease-directed group functions by supplying exogenous human N-acetylgalactosamine-6-sulfate sulfatase, represented by elosulfase alfa Consequently, these agents are utilized to manage mucopolysaccharidosis type IVA, also known as Morquio A syndrome, h...
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nih.gov
Mucopolysaccharidosis IVA (MPS IVA) treatment agents represent a therapeutic category composed of disease-specific therapies, primarily recombinant enzyme replacement therapy While not a standard formal FDA pharmacological class designation, this disease-directed group functions by supplying exogenous human N-acetylgalactosamine-6-sulfate sulfatase, represented by elosulfase alfa This mechanism facilitates the catabolism and cellular clearance of accumulated glycosaminoglycans, specifically kera...
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