Recently, Vtesse announced they had secured $17 million in additional funding to support their ongoing pivotal clinical trial of VTS-270 for treating Niemann-Pick type C1 disease. We talked to the CEO of Vtesse, Ben Machielse, about the study and the horrific impact this disease can have on a family. Read the full story here.
A Newborn Screen for NPC
A Washington University School of Medicine-led research team has developed a mass spectrometry-based screen for the cholesterol storage disorder Niemann-Pick disease type C in newborns. Read the full story here.
Novel Drug Delivery System May Offer New Hope
Researchers at Oregon State University and other institutions have discovered a type of drug delivery system that may offer new hope for patients with Niemann Pick Type C1 disease. No treatment currently exists for the disease, however, a compound that shows promise is now undergoing clinical trials, but it has major drawbacks. New findings, published today in Scientific Reports, outline the … Read More
- Page 2 of 2