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In an ongoing, open-label extension study of cerliponase alfa (Brineura; BioMarin, San Rafael, CA) for treatment of patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as Batten's disease, the rate of clinical decline continued to decrease over a 3-year period.
For 19 of the 23 treated patients in this study, there was no unreversed declines in the motor-language scale of more than 2 points. The mean ML scores 3.8 points better than natural history of the disease would predict.
Cerliponase alfa, a recombinant form of human tripeptidyl-peptidase 1 (TPP1), the enzyme deficient in patients with CLN2 disease, was approved by the Food and Drug Administration (FDA) to slow the loss of ambulation in pediatric patients 3 years of age and older.
"Every day, week, month, and year of maintaining clinical function, including language and mobility, is critical to children with CLN2 disease and their families," said Angela Schulz, MD, lead study investigator from the Department of Pediatrics, Children's Hospital, University Medical Center Hamburg-Eppendorf, Germany. "Following these children has allowed us to better understand the effect of the treatment over time, which contributes to the advancement in the standard of care."
Liana Rosenthal, MD, PhD
Serge Gauthier, CM, CQ, MD, FRCPC; and Pedro Rosa-Neto, MD, PhD
Winnie Pao, MD