ELAPRASE (idursulfase)
METABOLIC DISEASE ENZYME REPLACEMENT - MUCOPOLYSACCHARIDOSIS · MUCOPOLYSACCHARIDOSIS TYPE II
SHIRE US INC.
Drug class
Mucopolysaccharidosis II (MPS II) Treatment Agents
Mucopolysaccharidosis II (MPS II) treatment agents represent an indication-specific therapeutic group consisting primarily of lysosomal enzyme replacement therapies—such as recombinant human iduronate-2-sulfatase (idursulfase)—alongside emerging targeted modalities like blood-brain barrier-penetrating enzyme fusion proteins and investigational gene therapies. Read more about Mucopolysaccharidosis II (MPS II) Treatment Agents
2 drugs in this class 2 manufacturers
Drugs in Mucopolysaccharidosis II (MPS II) Treatment Agents
AVLAYAH (tividenofusp alfa-eknm)
METABOLIC DISEASE ENZYME REPLACEMENT - MUCOPOLYSACCHARIDOSIS · MUCOPOLYSACCHARIDOSIS TYPE II
DENALI THERAPEU
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Mucopolysaccharidosis II (MPS II) treatment agents represent an indication-specific therapeutic group consisting primarily of lysosomal enzyme replacement therapies—such as recombinant human iduronate-2-sulfatase (idursulfase)—alongside emerging targeted modalities like blood-brain barrier-penetrating enzyme fusion proteins and investigational gene therapies.
These agents function by supplying or restoring functional iduronate-2-sulfatase enzyme activity, which catabolizes and prevents the systemic accumulation of the glycosaminoglycans dermatan sulfate and heparan sulfate within cellular lysosomes.
Clinically, this class is utilized exclusively for the management of mucopolysaccharidosis II (Hunter syndrome), where it improves walking capacity, reduces hepatosplenomegaly, and targets progressive multi-systemic somatic and neurocognitive manifestations.
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primetherapeutics.com
Mucopolysaccharidosis II (MPS II) treatment agents represent an indication-specific therapeutic group consisting primarily of lysosomal enzyme replacement therapies—such as recombinant human iduronate-2-sulfatase (idursulfase)—alongside emerging targeted modalities like blood-brain barrier-penetrating enzyme fusion proteins and investigational gene therapies Clinically, this class is utilized exclusively for the management of mucopolysaccharidosis II (Hunter syndrome), where it improves walking ...
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mdpi.com
Mucopolysaccharidosis II (MPS II) treatment agents represent an indication-specific therapeutic group consisting primarily of lysosomal enzyme replacement therapies—such as recombinant human iduronate-2-sulfatase (idursulfase)—alongside emerging targeted modalities like blood-brain barrier-penetrating enzyme fusion proteins and investigational gene therapies
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nih.gov
Mucopolysaccharidosis II (MPS II) treatment agents represent an indication-specific therapeutic group consisting primarily of lysosomal enzyme replacement therapies—such as recombinant human iduronate-2-sulfatase (idursulfase)—alongside emerging targeted modalities like blood-brain barrier-penetrating enzyme fusion proteins and investigational gene therapies These agents function by supplying or restoring functional iduronate-2-sulfatase enzyme activity, which catabolizes and prevents the system...
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nih.gov
These agents function by supplying or restoring functional iduronate-2-sulfatase enzyme activity, which catabolizes and prevents the systemic accumulation of the glycosaminoglycans dermatan sulfate and heparan sulfate within cellular lysosomes
-
fda.gov
Clinically, this class is utilized exclusively for the management of mucopolysaccharidosis II (Hunter syndrome), where it improves walking capacity, reduces hepatosplenomegaly, and targets progressive multi-systemic somatic and neurocognitive manifestations
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