Current Research

There are researchers, scientists, academic centers, pharmaceutical companies, and advocacy organizations working today to improve the understanding and treatment 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 disease and from molecular biology research on the function of the CLN5 and MFSD8 genes. A variant in the CLN5 gene leads to CLN5 disease; a variant in the MFSD8 gene leads to CLN7 disease.

Ongoing Research

Gene therapy clinical trials are research studies that are designed to help to determine whether a gene therapy approach is safe and effective for individuals living with a disease.

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A natural history study is an observational study that collects information about the natural history of a disease in the absence of intervention, from disease onset throughout the lifespan of an individual.

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Learn how your child may be eligible to join a gene therapy clinical trial or a natural history study.

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. More information about Neurogene’s research pipeline can be found here.

Living Batten | Patient with CLN7, a rare neurological disease
Living Batten | Patient with late-infantile CLN7, a very rare neurological diseases (version 8)

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 ClinicalTrials.gov, a website that provides access to information on publicly and privately supported clinical studies for various diseases and conditions.

Searching “Batten disease” in the search bar on ClinicalTrials.gov 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.

Living Batten | Patient with late-infantile CLN7, a very rare neurological diseases (version 8)