Dosing & Uses
Alpha-Mannosidosis
Pending FDA approval for treatment of patients with alpha-mannosidosis (AM)
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Pharmacology
Mechanism of Action
Recombinant human alpha-mannosidase used as enzyme replacement therapy for alpha-mannosidosis (AM)
AM is a deficiency of the lysosomal enzyme, alpha-mannosidase, caused by pathogenic sequence variants in the MAN2B1 gene
This deficiency leads to accumulation of mannose-rich oligosaccharides, causing impaired cellular function and apoptosis, which conversely leads to significant and diverse adverse clinical manifestations
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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.
