What We Do
NKH Crusaders is committed to assisting families affected by NKH by offering patient advocacy through awareness initiatives, caregiver support, and funding vital research to advance NKH treatments.
Funding Research
Raising funds to further NKH Research in the United States
As a rare orphan disease, NKH relies heavily on funding from those directly affected, making our mission vital. Since 2010,H Crusaders has supported groundbreaking research by Dr. VanHove at the University of and Dr. Haldar Kasturi at the University of Notre Dame, focusing on medication and gene therapy, respectively. Through partnerships with other NKH organizations and dedicated parents, we are committed to raising the necessary funds for a cure that could change lives.
Funding the Cure
Patient Advocacy
The NKH Crusaders Patient Listening Session with the FDA in December 2023 was a pivotal moment in raising awareness and advocating for better treatments for NKH.
Eight families shared their firsthand experiences, offering powerful insights that deepened the FDA's understanding of this rare and complex disorder.
Hi, I'm Kristin,
I want to tell you about our son Thomas who has Non-Ketotic Hyperglycinemia or NKH. NKH, AKA glycine encephalopathy (GCE), is a rare, devastating, and incurable disease; with a very wide spectrum of severity. Most babies born with this disorder die shortly after birth. Before death, they go through a period of lethargy, weak crying, muscle weakness, and seizures. Many babies that are born with this disease die within the first 3 months of life. Those who do make it past infancy will live with various lifelong issues. There are several forms of NKH. We are lucky that our son has been diagnosed with atypical NKH, which is the mildest (and most uncommon) form.
No parent wants to learn that their child suffers from an incurable disease. With approximately 500 children living with NKH worldwide, NKH is a "rare and orphaned disease". Essentially, it's up to those directly effected by NKH who drive the research for a cure. Every year a child is lost to this devastating condition. Our families want to do everything possible to ensure that their children have a fighting chance for a normal, healthy life. In order to fulfill this dream, we are dedicated to funding the cure for NKH.
