velmanase alfa (Pending FDA Approval)

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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.