News

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

New Zealand researchers used computed tomography (CT) to measure and monitor brain alterations in sheep models of Batten disease, a study reports. Data revealed that brain atrophy starts in specific regions of the brain, called occipital lobes, spreads to the whole cortex, and eventually leads to a severe reduction…

A new system of vagus nerve stimulation (VNS) therapy was implanted in the first patient with drug-resistant epilepsy as part of a global registry launched by LivaNova to evaluate the treatment in a real-world setting. VNS therapy is recommended only for patients whose seizures — a common occurrence in Batten disease patients…

Regenxbio is developing a novel, one-time gene therapy, called RGX-181, for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease. The company expects to submit an investigational new drug (IND) application to the U.S. Food…

Husbandry conditions of mouse models of CLN3-Batten disease may influence research outcomes, such as potential disease biomarkers, a new study reports. Researchers highlight the need to standardize the environment where animals are maintained to identify robust biomarkers that might help develop therapeutics for Batten disease subtypes. The study,…

Two types of nerve cells — microglia and astrocytes — are dysfunctional and may contribute to neurodegeneration associated with different types of neuronal ceroid lipofuscinoses (NCLs, aka Batten disease), according to a new mouse study. However, the reasons behind these cells’ dysfunction are distinct in infantile NCL (…

The U.S. Food and Drug Administration has accepted a new drug application (NDA) for UCB’s Nayzilam (midazolam) nasal spray for the acute treatment of seizures including seizure clusters and acute repetitive seizures. Acceptance of this NDA could result in Nayzilam’s approval in the U.S. in early 2019, potentially…

Agitation and involuntary motor activity, which normally occurs in individuals who suffered traumatic brain injuries, also happens in patients with juvenile neuronal ceroid lipofuscinosis (JNCL, also known as Batten disease), according to researchers. The study, “Paroxysmal sympathetic hyperactivity in Juvenile neuronal ceroid lipofuscinosis (Batten disease),” was published in Autonomic…

Healx has raised $10 million to accelerate therapy discovery for rare diseases, including Batten disease.  The company uses artificial intelligence, pharmacology expertise, and patient engagement in an effort to give those with rare diseases a better quality of life. Balderton Capital, Europe’s major…

A group of experts has created a set of guidelines to best manage late infantile Batten disease, also known as CLN2.  Although effective strategies exist, clinicians currently lack common management guidelines to best respond to the disease’s symptoms. Multidisciplinary approaches and consensual practice guidelines are…