CLN1 Enzyme Replacement Therapy Shows Promise in Animal Models

An experimental enzyme replacement therapy (ERT) — one injected directly into the brain — was found to lessen motor dysfunction and nerve cell death in mouse and sheep models of infantile Batten disease, a new study reports. Collaborations Pharmaceuticals, known as CPI, recently received nearly $3 million…

Study: Drinking Acidified Water May Help Patients With Infantile Batten

Drinking acidified water prevented motor impairment and decreased the toxic accumulation of molecular debris in brain cells in a mouse model of CLN1 disease, also called infantile Batten disease, a recent study reports. “In this study, we demonstrated that acidified drinking water had beneficial effects in … a mouse…

Gene Therapy Prolongs Lifespan, Alleviates Symptoms of Mice With CLN6 Disease, Study Shows

A one-time treatment with an investigational gene therapy prolongs the lifespan in a mouse model of CLN6 disease, and ameliorates motor, learning, and memory impairments, a study shows. The therapy’s safety and efficacy is currently being evaluated in a Phase 1/2 trial (NCT02725580) in…

Motor Impairments Are Early Sign of Juvenile Batten Disease, Study Finds

In most patients with juvenile Batten disease, motor impairments are already apparent at the time of diagnosis and are not related to vision loss, a study finds. The findings of the study, “Motor function impairment is an early sign of CLN3 disease,” were published in Neurology.