Current Research

There are researchers, scientists, academic centers, pharmaceutical companies, and advocacy organizations working today to improve the understanding of Batten disease for a more hopeful tomorrow. Because the late infantile types of CLN5 and CLN7 are rare, much of the information currently available comes from the experiences shared by the individuals living with Batten disease1 and from molecular biology research on the function of the CLN5 and CLN7 genes. Research in mice and sheep, and a natural history study is soon to be underway.

Source: Field resource conducted by Neurogene and Ten Bridge Communications, August 2019.

How You Can Help the Medical Community
Better Understand Batten Disease

A natural history study is an important step to advance understanding of a disease. A natural history study for Batten disease (CLN5 and CLN7) is soon to be enrolling patients.

Enrollment for a Late Infantile Variant CLN5 and CLN7 Disease Natural History Study is Soon to Be Underway

For information about the natural history study, please contact:

What is a natural history study?

Why is a Batten disease natural history study important?

The Commitment of Neurogene to Batten Disease (CLN5 and CLN7)

In addition to sponsoring this educational website, Neurogene Inc. is a biotech company committed to developing life-changing medicines for patients and families affected by rare, devastating neurological diseases.

Neurogene is partnering with researchers and clinicians to gain a greater understanding of late infantile variants of CLN5 and CLN7 Batten disease. The latest partnering research can be found here.

Batten Disease Studies

A clinical study entails research involving humans with the intended goal of strengthening medical knowledge. There are two types of clinical studies: interventional and non-interventional. In interventional clinical trials, participants help researchers evaluate the efficacy and safety of investigational therapies that may have the possibility of treating a disease. Natural history studies are non-interventional, which means that there are no therapeutic interventions.

Studies on Batten disease are registered on, a website that provides access to information on publicly and privately supported clinical studies for various diseases and conditions. Enrollment for a Late Infantile Variant CLN5 and CLN7 Disease Natural History Study is soon to be underway.

Searching “Batten disease” in the search bar on will result in a list of upcoming trials that are not yet recruiting, trials that are currently enrolling, and trials that have concluded, including any published data resulting from completed studies.