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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…

Fundraising, storytelling, and advocacy are the focus of this year’s International Batten Disease Awareness Day, observed annually on June 9 to bring attention to the group of inherited neurological conditions thought to affect about 1 in every 100,000 individuals globally. The overarching aim of the worldwide event is to…

Treatment with the investigational therapy Batten-1 (miglustat) for 18 months, or about 1.5 years, stabilized motor symptoms in patients with juvenile Batten disease, according to the final results from a Phase 1/2 trial. The progression of motor symptoms was considerably slowed down, as evaluated using the modified Unified Batten Disease…

Note: This story was updated March 5, 2024, to clarify that Taysha Gene Therapies set aside the development of TSHA-118, along with other experimental gene therapies, to focus on another treatment for a different disorder. A patient with CLN1 disease, also known as infantile Batten disease, has received treatment…