Brineura (Cerliponase Alfa)

Brineura is recombinant human tripeptidyl peptidase 1 (TPP1) replacement enzyme to slow loss of walking ability in symptomatic pediatric patients aged 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Brineura is infused into the...

Brineura (Cerliponase Alfa)

Brineura is recombinant human tripeptidyl peptidase 1 (TPP1) replacement enzyme to slow loss of walking ability in symptomatic pediatric patients aged 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Brineura is infused into the...

Onpattro (Patisiran)

Onpattro (patisiran) is an intravenous RNA interference (RNAi) therapeutic agent for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. RNAi is a natural cellular process of gene silencing. Onpattro is designed to silence...