Amicus Therapeutics has gained global development and commercial rights to 10 gene therapy programs for lysosomal storage disorders, including three potential first-to-market therapies for Batten disease. This pipeline expansion resulted from the acquisition of Celenex, a clinical stage gene therapy company. “The in-licensing and acquisition of these gene therapy programs…
News
Exome sequencing, a technique that looks for gene mutations, is a valuable tool to diagnose pediatric neuronal ceroid lipofuscinoses, also known as Batten disease, according to a new case report. The study, “A novel MFSD8 mutation in a Russian patient with neuronal ceroid lipofuscinosis type 7: a case report,” was…
An animal study using a mouse model of CLN3 disease, the juvenile form of Batten disease, failed to identify any new biomarkers that could be used to track disease progression, which researchers say may be a result of variations in animal husbandry. The study, “Searching for novel biomarkers…
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…
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Researchers Use CT Scans to Monitor Long-term Brain Alterations in Sheep Models of Batten Disease
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…
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