There is currently no effective treatment for infantile neuronal ceroid lipofuscinosis, a neurodegenerative lysosomal storage disease that occurs in childhood. Here the authors show that a small molecule thioesterase-mimetic can alleviate neuropathology and extend lifespan in an animal model of the disease.
- Chinmoy Sarkar
- Goutam Chandra
- Anil B Mukherjee
