Empowering Families
and Professionals
The AHCF provides opportunities for collaboration, the latest in AHC literature and research education, and resources to empower AHC families and healthcare professionals as they care for individuals with AHC.
What are you looking for?
Find helpful information on the various categories below.
How We’re Making a Difference
Donating to the AHC Foundation allows us to fund the foremost research to end AHC, providing hope to AHC families around the world.
Recent News & Updates
Big News from Liu Lab – Gene Editing Project
Dear Friends and Families of the AHC Patient Community:
We want to update you on some exciting news related to our gene editing project.
Over the last several months, the Liu Lab has tested their gene editing strategy in AHC mice at the Jackson Laboratory. Preliminary results are encouraging.
The Liu team has found that treatment with prime editors can correct the Atp1a3 D801N mutation in the mouse brain and improve multiple clinically-relevant symptoms in D801N mice.
This is the first reported in vivo rescue of a neurological disease with prime editing.
This data is the first piece of evidence that prime editing may work as a therapeutic strategy in vivo in AHC. We are still many steps away from a therapy for AHC patients, but this result is a very positive first step on the long road ahead.
These findings are also the result of many years of effort from Drs. David Liu, Alex Sousa, and the Liu lab, and from Drs. Cat Lutz and Markus Terrey, and their colleagues at Jax. We are truly grateful to partner with such an incredible team.
Future experiments designed to edit the E815K mutation in mice will begin soon. The Liu team has made history. They have taken AHC to the front lines of cutting-edge experimental research.
AHC Update at Lurie Childen’s Hospital
Our Medical Advisory Board members, Dr. Sho Yano and Dr. Ken Silver, did a joint presentation on May 4, 2024, on the latest information on AHC related to ATP1A3 at Lurie Children’s Hospital for the Neurology department. They did a great job introducing the complexities of ATP1A3 and educating the staff about AHC. One topic of note – the time it takes to diagnose an AHC patient now is measured in weeks vs. the years it took when Dr. Silver started 20 years ago.
Upcoming Events
Family Meeting 2025
Exciting news coming soon! We have big plans for our 2025 family meeting. It’s a great time to meet a committed, wonderful group of people determined to find a cure. You’ll also experience in person an amazing and supportive community and learn about the latest research. Follow us on Facebook for the latest news.
AHCF relies entirely on volunteers
Every hour donated has a lasting impact on the lives of those with AHC! There are many ways to show your support, find out how you can get involved today.
Learn about our Research
AHCF is at the forefront of advancing cutting edge research to find a treatment and a cure for this one in a million disorder.