Brineura

Brineura is a medication used to treat a rare genetic condition called Batten disease, specifically a form known as late infantile neuronal ceroid lipofuscinosis Type 2 (CLN2). This disease affects the nervous system, causing progressive loss of skills and vision. Brineura works by replacing a missing enzyme that is crucial for breaking down certain fatty substances in the brain. Administered directly into the cerebrospinal fluid through a surgical port, it aims to slow down the disease's progression, improving patients' quality of life and helping maintain their abilities for a longer period.