Brineura (API Cerliponase alfa)

$600,000.00

Cerliponase alfa is an enzyme replacement therapy used to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.

Description

Buy Cerliponase alfa

Buy Cerliponase alfa is an enzyme replacement remedy for a particular shape of Batten disease. It changed into the first FDA-accepted treatment to slow lack of walking ability (ambulation) in symptomatic pediatric sufferers three years of age and older with overdue childish neuronal ceroid lipofuscinosis kind 2 (CLN2), additionally called tripeptidyl peptidase-1 (TPP1) deficiency. Intraventricular management of the drug permits widespread uptake into the mind.

Pharmacodynamics

Cerliponase alfa incorporates the energetic substance tripeptidyl peptidase-1 (rhTPP1), a recombinant human lysosomal exopeptidase which cleaves the N-terminal of tripeptides with a large substrate specificity. Cerliponase alfa slows the revolutionary decline in motor feature as a result of peculiar motor signalling inside the brain with the aid of restoring the regular levels and pastime of TPP1.

Mechanism of movement

The mature shape of enzyme contains five consensus N-glycosylation websites with high mannose, phosphorylated high mannose and complicated glycosylation structures. it’s far taken up through LINCL fibroblasts and translocated to the lysosomes through the Cation impartial Mannose-6-Phosphate Receptor (CI-MPR, also referred to as M6P/IGF2 receptor). Cerliponase alfa is activated inside the lysosome underneath low pH situations and the activated proteolytic form of rhTPP1 cleaves tripeptides from the N-terminus of stored proteins. Buy Cerliponase alfa

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Additional information

Quay

1 year annual supply