Skip to main content
BACK TO TOP
- NCLs constitute a group of the most common hereditary neurodegenerative Lysosomal Storage Diseases (LSDs)
- Commonly known as Batten disease;
- Mutations in 13 different genes cause various types of NCLs;
- The infantile NCL (INCL) is one of the most devastating among all NCLs;
- INCL is caused by inactivating mutations in the CLN1 gene, which encodes palmitoyl-protein thioesterase-1 (PPT1);
- Currently, there is no effective treatment for any of the NCLs.