Batten Disease Treatment Includes Early Detection And Gene Therapy

 

Batten disease treatment is a search for a way to treat or prevent symptoms that occur in the body and brain when certain mutations affect lysosomes. These organs are responsible for breaking down waste in cells and recycling them for use elsewhere in the body. When a faulty gene makes the wrong protein, lysosomes cannot do their job and build up cellular waste that can cause problems throughout the body.

The earliest signs of the condition may be vision loss, especially when a child is young. Batten Disease Treatment can be detected by a routine eye exam or special tests of the eyes, such as visual-evoked response (VER) and electroretinograms (ERG).

Along with vision problems, a weakened immune system may also occur in people with Batten disease. One type of therapy uses a medication that blocks a type of immune system cells called macrophages. This may help reduce the buildup of fatty proteins and other materials in the brain that can cause seizures and psychiatric problems.

Seizures and psychiatric symptoms are often controlled or reduced with anticonvulsant drugs, and other medical problems can be managed as they develop. Physical and occupational therapy can also help children retain motor functioning as long as possible.

Some forms of Batten disease can be treated by injecting a gene into the brain, which will correct the problem with lysosomes. This approach is called gene therapy. It has been shown to be effective in other inherited lysosomal storage disorders, and may prove useful for NCL patients. However, gene therapy requires strict inclusion and exclusion criteria in order to protect the safety of participants.

There are four major types of Batten disease: CLN1 disease, CLN2 disease, late infantile neuronal ceroid lipofuscinosis (CLN2) disease, and CLN6 disease. Each form has its own unique set of symptoms.

In most cases, Batten disease is caused by a mutated gene that leads to the buildup of a substance called lipofuscins in the body. This can cause problems with the nerves that control movement and sense of things, such as touch and sound.

These deposits can also block signals to and from the brain that tell the body when it needs food or fluids, as well as send messages about pain and temperature. These problems can lead to deformities and other health complications, including blindness.

Early diagnosis is important to preventing complications and death from Batten disease. The condition usually starts in the child’s first few months of life, and symptoms can quickly get worse.

A clinical-stage company Neurogene Inc., declare that their Investigational New Drug application for NGN-401 has been cleared by the US FDA, in January 2023.