CLN5 disease, a form of late infantile Batten disease, disrupts fatty molecule balance and autophagy (the waste disposal system…
Articles by Marta Figueiredo, PhD
Two dozen new mutations in the CLN6 gene were identified in nearly 100 people, mostly from the Middle East and North…
The number of activated microglia — the brain’s resident immune cells — are significantly increased by the time seizures start…
The layers of the retina — the part of the eye that’s damaged in Batten disease — are differently…
During the COVID-19 pandemic in England, treatment with Brineura (cerliponase alfa) was considered “essential” for children with neuronal…
Mutations in the CLN6 gene — which have been associated with different forms of Batten disease — may lead…
An international team of experts has developed a set of consensus-based, family-informed recommendations for the diagnosis and best care management…
A screening of U.S.-approved therapies revealed that tamoxifen, an oral cancer therapy, is able to effectively reduce the accumulation…
A gene therapy designed to deliver a working version of the CLN8 gene — the one mutated in people with…
Two years of treatment with Brineura (cerliponase alfa) safely and effectively delayed the onset of late-infantile Batten disease in…