News

Structural and functional changes in the retinal pigment epithelium (RPE) — a cell layer in the eye that nourishes the light-sensing cells — may be behind the progressive vision loss seen in juvenile Batten disease, or CLN3 disease, according to a recent study. Using lab-grown patient-derived and healthy RPE…

Neurogene has decided to discontinue its research program focusing on CLN7, a form of late-infantile Batten disease, including the CLN7 portion of its natural history study. The goal of this natural history study (NCT03822650), focused on patients with CLN7 and CLN5 forms of Batten disease, is to…

Some cases of Batten disease may present with clinical features of Rett syndrome, including Rett’s hallmark repetitive hand movements, according to a recent case report concerning two young girls. Features that helped distinguish Batten disease in these girls included vision loss, early brain shrinkage (atrophy), and evidence of…

Longitudinal MRI imaging in naturally-occurring sheep models of CLN5 and CLN6 disease — two types of Batten disease — revealed early and progressive brain volume loss, a study showed. A feature of this atrophy was the loss of gray matter and an increase in volume of the cerebrospinal fluid…

A single intravitreal injection — into the vitreous cavity, in the back of the eye — of a gene therapy was found to preserve the function and structure of the retina in a dog model of late infantile Batten disease. Eye inflammation was…

Treatment for up to two months with Batten-1 — Theranexus’ novel therapy for juvenile Batten disease, also known as CLN3 disease — was found to be generally safe and well-tolerated in patients ages 17 and older. That’s according to early data from an ongoing two-year Phase 1/2…

Alterations in producing, transporting, and degrading proteins, as well as in neuronal activity, are likely involved in juvenile Batten disease, according to data from lab-grown neurons from a patient with the most common disease-causing mutation. These findings point to previously unknown mechanisms that may lead to neurodegeneration among patients…

Both of Regenxbio‘s gene therapy candidates for neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as late infantile Batten disease, have advanced to in-human testing, the company reported. These clinical trials will evaluate the one-time experimental gene therapies in patients with the inherited disease, which typically occurs…

Researchers have proposed an alternate classification model for Batten disease types based on the site first affected by each type and how it progresses. They tested their hypothesis in two of the most common Batten types. A comprehensive analysis of published data so far showed infantile Batten disease…

Female mice with Batten disease caused by a mutation in the Cln8 gene had greater disease severity, including worse behavioral deficits and a shorter lifespan, than their male counterparts, a study has found. These female mice also had more activation in the brain of microglia, a type of cell that drives…