Steve Bryson, PhD,  science writer—

A new system that delivers Brineura (cerliponase alfa) into the brains of people with CLN2 disease, also known as late infantile Batten disease, via a port implanted in the chest provides a more stable and well-tolerated alternative to scalp-based infusions, a case series reports. None of the seven…

Sex-specific differences in sleep disturbances have been discovered in a mouse model of juvenile Batten disease, according to the first study of its kind. During the light period, when mice are primarily asleep, male mice with juvenile Batten-like disease spend less time asleep than healthy mice, while diseased female…

Drinking acidified laboratory water improved motor performance, restored muscle strength, prevented tremors, and extended survival in a mouse model of late infantile Batten disease, a study reports. Researchers also noted pronounced changes in gut bacterial composition in Batten mice given acidified drinking water, which might have contributed to its…

Researchers have developed a mouse model of juvenile Batten disease that better reflects the survival outcomes of human patients, according to a new study. While mice without the gene that carries instructions for the CLN3 protein recapitulate many juvenile Batten features, these mice have milder disease and a much…

Gene therapy was able to reduce the signs and symptoms of late infantile Batten disease in a mouse model carrying a common disease-causing mutation, a study shows. These findings support Regenxbio’s ongoing gene therapy program, which includes RGX-181, currently being tested as part of a single-patient study…

Changes in the levels of certain metabolites in the brain among people with juvenile Batten disease, also called CLN3 disease, are significantly associated with multiple measures of disease severity, a study showed. Age accounted for many of these metabolite changes. Still, the levels of some brain metabolites — the…

A lack of the CLN3 protein — the underlying cause of juvenile Batten disease — impairs the function of stress granules (SGs), which are formed to promote cell survival under stressful conditions, according to a cell-based study. CLN3-deficient cells also had lower metabolic rates and were impaired in their…

Regenxbio says it will test RGX-381, its investigational gene therapy for eye problems associated with neuronal ceroid lipofuscinosis type 2 (CLN2) disease, in a first-in-human Phase 1/2 clinical trial that remains on track to begin in the first half of this year. The trial will evaluate the one-time…

Neurogene has decided to discontinue its research program focusing on CLN7, a form of late-infantile Batten disease, including the CLN7 portion of its natural history study. The goal of this natural history study (NCT03822650), focused on patients with CLN7 and CLN5 forms of Batten disease, is to…

Researchers have proposed an alternate classification model for Batten disease types based on the site first affected by each type and how it progresses. They tested their hypothesis in two of the most common Batten types. A comprehensive analysis of published data so far showed infantile Batten disease…