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Tern Therapeutics is partnering with Andelyn Biosciences to develop its investigational gene therapy that targets vision loss in children with CLN2 Batten disease, also known as late-infantile Batten disease. Andelyn, a contract development and manufacturing organization specializing in cell and gene therapy, will participate in the…
A personalized treatment called Zebronkysen led to gains in function for twin girls with juvenile Batten disease in a clinical study, surpassing the expectations of researchers who had hoped merely to slow disease progression. “We did not expect to see improvement in addition to a slowing of decline,” Yael…
Treatment for approximately one year with Batten-1 (miglustat) — an experimental oral small molecule developed by Theranexus — in individuals with juvenile Batten disease, also known as CLN3 disease, resulted in significantly slower vision loss compared with untreated patients, according to new data from a real-world study. The…
The global Batten disease community will come together Monday, June 9, to connect and spread awareness about the rare genetic disorder. To mark International Batten Disease Awareness Day, the Batten Disease Support and Research Association (BDSRA) is planning a virtual 5K in hopes of raising $100,000 to help…
A new system that delivers Brineura (cerliponase alfa) into the brains of people with CLN2 disease, also known as late infantile Batten disease, via a port implanted in the chest provides a more stable and well-tolerated alternative to scalp-based infusions, a case series reports. None of the seven…
Blood tests to look for a specific type of abnormal immune cell called vacuolated lymphocytes may help diagnose juvenile Batten disease, a report showed. The study, “Vacuolated lymphocytes: a diagnostic biomarker for CLN3-related Batten disease,” was published in the Canadian Journal of Ophthalmology. Juvenile Batten disease…
Sex-specific differences in sleep disturbances have been discovered in a mouse model of juvenile Batten disease, according to the first study of its kind. During the light period, when mice are primarily asleep, male mice with juvenile Batten-like disease spend less time asleep than healthy mice, while diseased female…
Gene therapy targeting nerve cells in the gut improved nerve cell survival and intestinal function — specifically that of the bowel — while also increasing survival rates in a mouse model of Batten disease, a new study by researchers at Washington University School of Medicine (WashU Medicine) in St.
People with juvenile Batten disease treated with the small molecule Batten-1 (miglustat) for 18 months in a clinical trial, and who continued with treatment outside the study, showed no signs of deteriorating vision, a common disease symptom, the company developing Batten-1, Theranexus, announced. All patients completed…
A new mutation in the KCTD7 gene was identified in a boy as the cause of CLN14 disease, a rare form of infantile-onset Batten disease. The report, “A novel pathogenic variant in the KCTD7 gene in a patient with neuronal ceroid lipofuscinosis (CLN14): a case report and review of the…
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