Brineura is a medication that has been approved by the FDA for the treatment of a rare genetic disorder known as late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. This condition is characterized by the buildup of lipofuscin in the cells of the brain and nervous system, leading to a progressive decline in neurological function.

Brineura works by replacing the deficient enzyme, tripeptidyl peptidase 1 (TPP1), which is responsible for breaking down the lipofuscin that accumulates in the cells. By providing the missing enzyme, Brineura helps to reduce the buildup of lipofuscin and slow the progression of the disease.

It is important to note that Brineura is administered directly into the fluid surrounding the brain through a surgically implanted device called a reservoir and catheter system. This delivery method allows the medication to reach the affected cells in the brain and provide targeted treatment.

As with any medication, Brineura may cause side effects in some patients. These can include fever, vomiting, seizures, and irritability. It is important to discuss any concerns or potential side effects with your healthcare provider before starting treatment with Brineura.

Overall, Brineura represents a significant advancement in the treatment of CLN2 disease and offers hope to patients and families affected by this devastating condition. If you or a loved one has been diagnosed with CLN2 disease, I encourage you to speak with your healthcare provider about whether Brineura may be a suitable treatment option for you.