VTS-270 for Niemann-Pick disease type C1

February 2017
Technology Description:

VTS-270 is a new drug that is injected into the spine to treat Neimann-Pick disease, which is a rare genetic disorder that causes accumulation of fats in cells throughout the body, including the liver and the brain. It may lead to balance and learning difficulties and most patients do not survive beyond age 25. Some studies have suggested that VTS-270 may help patients with Neimann-Pick disease to have better quality of life.