News

Blood tests to look for a specific type of abnormal immune cell called vacuolated lymphocytes may help diagnose juvenile Batten disease, a report showed. The study, “Vacuolated lymphocytes: a diagnostic biomarker for CLN3-related Batten disease,” was published in the Canadian Journal of Ophthalmology.  Juvenile Batten disease…

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…

Gene therapy targeting nerve cells in the gut improved nerve cell survival and intestinal function — specifically that of the bowel — while also increasing survival rates in a mouse model of Batten disease, a new study by researchers at Washington University School of Medicine (WashU Medicine) in St.

People with juvenile Batten disease treated with the small molecule Batten-1 (miglustat) for 18 months in a clinical trial, and who continued with treatment outside the study, showed no signs of deteriorating vision, a common disease symptom, the company developing Batten-1, Theranexus, announced. All patients completed…

A new mutation in the KCTD7 gene was identified in a boy as the cause of CLN14 disease, a rare form of infantile-onset Batten disease. The report, “A novel pathogenic variant in the KCTD7 gene in a patient with neuronal ceroid lipofuscinosis (CLN14): a case report and review of the…

With its RareCare Patient Assistance Program, the National Organization for Rare Disorders (NORD) helps to cover healthcare costs that aren’t paid by health insurance — and the program is available to people with Batten disease in the U.S. who may be struggling to pay bills directly related to…

Measuring how unconscious brain signals respond to different sounds may help track neurocognitive decline in people with juvenile Batten disease, a new study suggests. The study, “Assessing the integrity of auditory sensory memory processing in CLN3 disease (Juvenile Neuronal Ceroid Lipofuscinosis (Batten disease)): an auditory evoked…

The University of Rochester Batten Center (URBC) was designated a Center of Excellence by the Batten Disease Support, Research, and Advocacy (BDSRA) Foundation in recognition of its efforts at advancing care and research for Batten disease. The BDSRA Center of Excellence Program seeks to improve outcomes in Batten…

The enzyme replacement therapy Brineura (cerliponase alfa) is now approved by the U.S. Food and Drug Administration (FDA) as a treatment for CLN2 disease, also known as late infantile Batten disease, in children of all ages. The FDA previously approved Brineura to slow the loss of walking or crawling…

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…