Author Archives: Marisa Wexler MS

An inflammatory environment driven by brain cells called microglia may act to prevent the brain from repairing itself in Batten disease, according to a new study done in sheep chimeras. The study, “Aggregation chimeras provide evidence of in vivo intercellular correction in ovine CLN6 neuronal ceroid…

A 13-year-old girl experiencing unexplained seizures and no other apparent symptoms was ultimately diagnosed with juvenile Batten disease, according to a recent report. This “is probably the first case report of [juvenile Batten disease] with only seizures as presentation,” the researchers wrote. Titled “Batten’s Disease: A…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Parents of children with juvenile Batten disease face many challenges, but may find value in small moments of joy, a new study highlights. Juvenile Batten, also called CLN3 disease, is the most common type of the inherited neurodegenerative disorder. Usually starting between ages 5 to 7 with vision…

Mutations in the gene CLN5, which cause a form of late infantile Batten disease, disrupt the function and movement of cellular compartments called lysosomes, the so-called “recycling centers” of cells. That’s according to the study “Deficiency of the Lysosomal Protein CLN5 Alters Lysosomal Function and Movement,” which…

Children with CLN5, a type of late infantile Batten disease, are now being recruited for a first-in-human clinical trial testing NGN-101, Neurogene‘s experimental gene therapy for the rare neurological condition. The trial will be conducted at the University of Rochester Medical Center (URMC), in upstate New York, the company …

In children with late infantile Batten disease, degeneration in a specific part of the eye’s retina called the parafovea occurs before other parts of the retina are affected, a study reported. The study, “Automated Retinal Layer Segmentation in CLN2-Associated Disease: Commercially Available Software Characterizing a Progressive…

Enrollment in a clinical trial testing NGN-101 — an experimental gene therapy for CLN5, a type of late-infantile Batten disease — is expected to start early next year at the University of Rochester Medical Center (URMC). “This trial will move research forward in developing a potentially disease-modifying treatment for…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Beyond Batten Disease Foundation (BBDF) has struck a deal with Actelion Pharmaceuticals to help further the development of BBDF-101, an investigational treatment for juvenile Batten disease. Under the terms of the new agreement, Actelion (a Janssen Pharmaceutical Company of Johnson & Johnson) will provide a component of…