| abstract |
There is currently no effective cure or treatment for neurodegenerative diseases associated with progranulin (PGRN) deficiency, such as frontotemporal dementia (FTD). FTD is a fast-progressing disease, with affected patients usually dying 2-5 years after clinical diagnosis, and is the leading cause of non-Alzheimer dementias. The present application relates to methods and uses for the treatment of neurodegenerative diseases associated with PGRN deficiency such as FTD. Such methods and uses are based on the administration of agents that increase sphingolipid (SL) levels in neural cells from the subject, or of AchR and/or PKC inhibitors such as rottlerin, rivastigmine, or analogs or pharmaceutically acceptable salts thereof. |