ALDURAZYME (laronidase)
METABOLIC DISEASE ENZYME REPLACEMENT - MUCOPOLYSACCHARIDOSIS · MUCOPOLYSACCHARIDOSIS TYPE I
GENZYME
Drug class
Mucopolysaccharidosis I (MPS I) Treatment Agents
Mucopolysaccharidosis I (MPS I) treatment agents represent an indication-specific therapeutic classification primarily defined by enzyme replacement therapies developed to manage alpha-L-iduronidase deficiency. Read more about Mucopolysaccharidosis I (MPS I) Treatment Agents
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Mucopolysaccharidosis I (MPS I) treatment agents represent an indication-specific therapeutic classification primarily defined by enzyme replacement therapies developed to manage alpha-L-iduronidase deficiency.
Although the phrase functions as a disease-specific formulary category rather than a formal pharmacologic class name, its primary approved agent is recombinant human alpha-L-iduronidase (laronidase), which works by facilitating the cellular catabolism and clearance of accumulated glycosaminoglycans such as dermatan sulfate and heparan sulfate.
These agents are indicated for the management of the MPS I clinical spectrum—including Hurler, Hurler-Scheie, and moderate-to-severe Scheie syndromes—to improve non-neurological systemic manifestations, joint mobility, and pulmonary capacity.
Because intravenously administered enzymes cannot cross the blood-brain barrier, they are often utilized alongside or sequentially with hematopoietic stem cell transplantation for severe disease phenotypes.
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aldurazyme.com
Mucopolysaccharidosis I (MPS I) treatment agents represent an indication-specific therapeutic classification primarily defined by enzyme replacement therapies developed to manage alpha-L-iduronidase deficiency Although the phrase functions as a disease-specific formulary category rather than a formal pharmacologic class name, its primary approved agent is recombinant human alpha-L-iduronidase (laronidase), which works by facilitating the cellular catabolism and clearance of accumulated glycosami...
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nih.gov
Mucopolysaccharidosis I (MPS I) treatment agents represent an indication-specific therapeutic classification primarily defined by enzyme replacement therapies developed to manage alpha-L-iduronidase deficiency Although the phrase functions as a disease-specific formulary category rather than a formal pharmacologic class name, its primary approved agent is recombinant human alpha-L-iduronidase (laronidase), which works by facilitating the cellular catabolism and clearance of accumulated glycosami...
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molinahealthcare.com
Although the phrase functions as a disease-specific formulary category rather than a formal pharmacologic class name, its primary approved agent is recombinant human alpha-L-iduronidase (laronidase), which works by facilitating the cellular catabolism and clearance of accumulated glycosaminoglycans such as dermatan sulfate and heparan sulfate These agents are indicated for the management of the MPS I clinical spectrum—including Hurler, Hurler-Scheie, and moderate-to-severe Scheie syndromes—to im...
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chop.edu
Because intravenously administered enzymes cannot cross the blood-brain barrier, they are often utilized alongside or sequentially with hematopoietic stem cell transplantation for severe disease phenotypes
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