CIBER

Therapeutic use of acid ceramidase in the treatment of Niemann-Pick type C disease

Biodrugs/ Drugs
Blood and Lymphatic systems
Cancer
The objective is to administer recombinant acid ceramidase (ACDase) as a new treatment in type C Niemann-Pick disease. ACDase, whose expression is depressed in NPC disease, is capable of suppressing the induction of STARD1.
1Non Regulatory Preclinical