Magdalena Kegel,  —

In rare cases, a type of Batten disease may appear at birth, write researchers in a case report, describing a newborn with severe brain damage. The research team at the University Hospitals of Geneva said the disease — also called congenital neuronal ceroid lipofuscinosis type 10 (CLN10) — was…

Abnormal processing of the trace element manganese may impact the onset or progression of Batten disease, according to a researcher at the University of Melbourne in Australia. In a review titled “Manganese in manganism, Parkinson’s disease, Huntington’s disease, amyotrophic lateral sclerosis, and Batten disease: A narrative review,”…

People with Batten disease would likely benefit from early educational interventions that allow them to make up for their lost eyesight and speech with alternative communication skills — even before they are actually needed — according to a report from the Juvenile Neuronal Ceroid Lipofuscinosis and Education Project (2014 – 2017).

Batten disease may be caused by the loss of synaptic connections between neurons in the brain, according to a study that detailed abnormalities in the brains of mice, lacking the Cln3 gene. These mice also lacked a certain type of neuron crucial for building neural networks. Researchers at Jena University…

Yeast models of Batten disease and other lysosomal storage disorders could speed up the discovery of treatments by overcoming the restraints of animal models of the disease, a group of researchers argues in the journal Microbial Cell. Their article describes how a research approach that starts in yeast and ends…

Although Brineura (cerliponase alfa) is now approved both in Europe and the U.S. to treat CLN2 type Batten disease, its clinical trial path is far from over. As the approval rested on a small Phase 1/2 trial, an ongoing extension study (NCT02485899) is gathering additional safety and efficacy data. Meanwhile, the…

Mouse experiments with a gene therapy for juvenile Batten disease indicate that lower activation of the mutated CLN3 gene are more beneficial than high levels. While the research team was the first to show that the specific gene delivery method can be used for treating Batten disease, their findings illustrate…

Patients with a specific type of Batten disease now have an approved treatment, with the U.S. Food and Drug Administration’s (FDA) recent approval of Brineura (cerliponase alfa) for use in late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Brineura is an enzyme replacement therapy, providing CLN2 patients with a lab-produced…