Miglustat, an investigational oral medication currently in early clinical testing for Batten disease, was tolerated well in a small study that evaluated the therapy in six people with juvenile Batten disease. Trial data indicate that the miglustat-treated patients had a slower rate of progression in physical impairment than…
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LTS-101, a one-time gene therapy designed to treat children with late infantile Batten disease, is poised to enter clinical testing, according to an update from its developer, Latus Bio. Latus announced that the U.S. Food and Drug Administration (FDA) has granted clearance to its investigational new drug (IND)…
Brain abnormalities in juvenile Batten disease can vary according to biological sex and age, a new study in mice has found. The research, which focused on brain activity in response to sound, found that female Batten mice had brain activity abnormalities that were evident at an early age and…
New research of mouse models of juvenile Batten disease finds that disruptions in brain activity emerge soon after birth. The findings suggest treatments for this form of Batten disease, such as gene therapies, are likely most effective when given as early as possible. The study, “…
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…
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