CLN3, the protein missing in people with juvenile Batten disease, is needed for a cellular process normally taking place…
Articles by Joana Carvalho, PhD
AT-GTX-502, Amicus Therapeutics’ investigational gene therapy for juvenile Batten disease, has been well-tolerated and is showing a potential…
Small vesicles naturally produced by certain types of immune cells may be used as carriers to deliver TPP1 — the…
Signs of mild atrophy, or shrinkage, in certain areas of the brain, including the cerebellum — a region involved…
A reduction in the ability of the CLN6 protein to prevent other proteins from clumping together and forming aggregates can…
Brineura (cerliponase alfa), the first and only approved treatment for CLN2 disease, or late infantile…
Scientists have identified a series of proteins that may be useful biomarkers of disease progression for different types of…
Treatment with a type of complex fat molecule called galactosylceramide increased lifespan, improved behavioral outcomes and eased neuronal loss in…
A one-time treatment with an investigational gene therapy prolongs the lifespan in a mouse model of CLN6 disease,…
Brain-directed gene therapy is a feasible therapeutic strategy to prevent neurodegeneration associated with CLN6 disease, a mouse study says.