What is CLN2 Batten Disease?
CLN2 Batten disease is a rare, inherited and progressive neurological condition that primarily affects young children.
It is one of a group of conditions known as Batten disease, or neuronal ceroid lipofuscinosis (NCL).
CLN2 is caused by changes in the TPP1 gene, which result in the body not producing enough of an important enzyme called TPP1. Without this enzyme, waste materials build up inside cells, particularly cells in the brain and nervous system, causing progressive damage.
There is currently no cure for CLN2 Batten disease. However, an enzyme replacement therapy called Brineura (cerliponase alfa) can help slow the progression of certain aspects of the disease.
Behind every diagnosis is a child and a family navigating an incredibly rare and life-changing journey. Raising awareness helps bring greater understanding, support and hope for children living with Batten disease and their families.
Children with CLN2 often develop typically during their early years before symptoms begin, commonly between the ages of 2 and 4. Early signs can include speech or language regression and seizures. As the disease progresses, children can gradually lose skills they have already developed, including their ability to walk, talk, eat and see
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