Search Results for: ceroid lipofuscinosis type 2


ABO-202 is an investigative, one-time gene therapy for a type of Batten disease called CLN1, or rare infantile neuronal ceroid lipofuscinosis. ABO-202 is produced by Abeona Therapeutics and is currently in ... Read more

Batten Disease Types

Batten disease is a severe, heritable nervous system disorder. Although originally used to describe only the form of the disease that begins between the ages of 5 and 10, ... Read more


PLX-200 is an investigational oral small molecule that was originally developed to lower cholesterol, and is being repurposed by Polaryx Therapeutics to potentially treat a type of Batten disease called late infantile neuronal ceroid ... Read more