{"id":113,"date":"2024-11-20T16:00:55","date_gmt":"2024-11-20T16:00:55","guid":{"rendered":"https:\/\/www.brineura.com\/en-us\/?page_id=113"},"modified":"2026-03-12T13:44:48","modified_gmt":"2026-03-12T13:44:48","slug":"treatment","status":"publish","type":"page","link":"https:\/\/www.brineura.com\/en-us\/treatment\/","title":{"rendered":"Treatment"},"content":{"rendered":"
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Brineura®<\/sup> (cerliponase alfa) is the only treatment that directly addresses the underlying cause of CLN2 disease by helping to replace the TPP1 enzyme1<\/sup><\/h1>\n

Brineura®<\/sup> (cerliponase alfa<\/span>)<\/span> is a type of treatment called enzyme<\/span> replacement therapy. Brineura helps to replace the TPP1 enzyme, the enzyme that is missing or not working properly in children with CLN2 disease<\/span>.1<\/sup><\/p>\n <\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Brineura was evaluated in 24 children with CLN2 disease in a clinical study and its follow-up study1<\/sup><\/h2>\n

Each child’s ability to walk, with or without assistance, was evaluated over approximately 2 years. Their results were compared with records of untreated patients who experienced the rapid, predictable loss of mobility that occurs in CLN2 disease<\/span>.1<\/sup><\/p>\n <\/div>\n\n

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Children in the study ranged from 3 to 8 years old. The average age at first symptom was 3.4 years (ranged from 2.5 to 6.3 years)1,2<\/sup>\n<\/h3>\n <\/div>\n<\/div>\n\n
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Children in the extension study<\/span> were evaluated at approximately 2 years; the study is still ongoing1<\/sup><\/h5>\n <\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n
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The CLN2 Clinical Rating Scale was used to measure how Brineura works1<\/sup><\/h2>\n

Children’s ability to walk and crawl was measured using the CLN2 Clinical Rating Motor Domain Scale<\/span><\/span>.<\/strong> Scores range from 3 (normal) to 0 (loss of walking\/crawling abilities).1<\/sup> Within each score, there may be some differences in the way each child shows their ability to walk or crawl.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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*In some children, walking abilities were never completely normal and were rated as a 2.\n

<\/p>\nAdapted from: Steinfeld R, et al. Am J Med Genet<\/i>. 2002:112:347-354.\n<\/figcaption>\n <\/figure>\n <\/div>\n <\/div>\n<\/div>\n\n

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Brineura helped maintain children’s ability to walk, with or without assistance, over approximately 2 years of treatment1<\/sup><\/h2>\n\n\n

In the clinical study, decline was defined as a sustained drop of 2 points or a score of 0 on the CLN2 Clinical Rating Scale<\/span>.1<\/sup><\/p>\n\n\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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