Can Batten disease be treated?
Currently, there is only one FDA-approved treatment. It’s only for children with CLN2. Children receive infusions of a medicine called cerliponase alfa (Brineura®) every two weeks. Providers inject the medicine directly into the fluid surrounding the child’s brain. The medicine slows down the loss of the ability to crawl or walk (ambulation). However, it does not slow down other symptoms of the disorder.
Researchers are studying many promising treatments for Batten disease. These include medications, stem cell transplants and gene therapy to replace the faulty gene. Until other treatments for Batten disease are available, healthcare providers focus on treating the symptoms of the disorder.
Children with Batten disease often have a team of providers to address the many symptoms. Your team of providers may prescribe medications to control seizures or drugs to prevent hallucinations. Physical therapy and occupational therapy can help relieve muscle spasticity in some children and retain mobility for a longer time.