News

Stem cell models of lysosomal storage disorders (LSDs), including Batten disease (BD), could significantly add to knowledge of disease biology and help develop improved therapies, a new review highlights. The study, titled “Induced pluripotent stem cell models…

The fat-lowering therapy gemfribrozil decreased disability and increased the lifespan of mice with a form of Batten disease that strikes children, according to a preclinical-trial study. The finding suggests the high-cholesterol drug could be used to treat children with Batten, which scientists call Neuronal Ceroid Lipofuscinosis, or NCL. The research also indicated…

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…

 Brineura (cerliponase alfa), by BioMarin, is now approved in Europe to treat neuronal ceroid lipofuscinosis type 2 (CLN2), one of a group of disorders known as Batten disease. The therapy covers all ages from birth. The marketing authorization granted by the European Commission (EC) follows a positive review by…

Treatment with anti-inflammatory drugs fingolimod (Gilenya) and teriflunomide (Aubagio) may potentially fight brain inflammation and neuronal death in patients with neuronal ceroid lipofuscinoses (NCLs or CLNs), according to new reserch. CLNs are a group of disorders of which Batten disease is the most common. Aubagio, marketed by…

Lopid (gemfibrozil), an FDA-approved lipid-lowering drug, was found to improve life expectancy and motor function in mice with late infantile neuronal ceroid lipofuscinosis (late infantile NCL, or LINCL), which belongs to a group of disorders collectively referred to as Batten disease. The study, “Gemfibrozil, Food and Drug Administration-approved lipid-lowering drug,…

Compounds that inhibit the effects of enzymes linked to brain inflammation improve movement in mice with both early and advanced Batten disease, according to a study. The compounds are called PDE4 (3 phosphodiesterase‐4) inhibitors because they suppress PDE4 enzyme activity. University of Nebraska Medical Center researchers said their study was…

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…

The Batten Disease Support and Research Association (BDSRA) will hold its 2017 Family Conference in July, bringing together patients and families touched by Batten disease. The on July 6–9 event will take place at the Wyndham Grand Pittsburgh Downtown. The conference joins family members, children and researchers to…

Abeona Therapeutics‘ ABO-201 reduced nerve inflammation and improved movement in mice with juvenile Batten disease (JBD), according to a preclinical trial study. Scientists at Abeona designed ABO-201 to overcome the effects of the CLN3 genetic mutation that is responsible for the disease. A single injection led to the correct form of the gene being delivered…