2017 APMRF Funded Grants

We are excited to announce the 2017 APMRF funded grants.  Please go to this LINK to see the list of funded researchers and the focus of their research. Research areas range from fundamental science of the NPC pathway, the development of new therapeutics for NPC disease, and also the support of new gene-therapy efforts.  We are also very excited that … Read More

Conversation with CEO of Vtesse about Niemann-Pick Type C

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