22 June 2021
Two years of treatment with Brineura (cerliponase alfa) safely and effectively delayed the onset of CLN2 disease in a 23-month-old boy who had not exhibited symptoms when he started treatment, a case study shows.
The same treatment slowed disease progression in his older sister, who received the therapy after symptom onset at 47 months (nearly 4 years) of age.
While preliminary, these findings suggest that Brineura can slow disease progression when CLN2 disease is already established, and also delay disease onset before symptoms show (pre-symptomatic).
The case study, “Presymptomatic treatment of classic late-infantile neuronal ceroid lipofuscinosis with cerliponase alfa,” was published in the Orphanet Journal of Rare Diseases.