NKH Research
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Advancing NKH Research Together
As we come to the end of another year, we’d like to thank everyone who has supported Joseph’s Goal during 2025.
We’ve recently reached the 13-year anniversary of our charity…and look how far we’ve come! We remain committed to the work of Dr Greene at UCL.
Key Progress Highlights
Gene Therapy Progress
Gene therapy has successfully reduced harmful glycine levels in the brain in NKH models, marking a major step towards future treatments.
Research Accelerated
Funding from Joseph’s Goal has accelerated NKH research by 12–18 months, helping critical studies move forward faster.
Pathway to Clinical Trials
Plans are in place for final safety testing, medicine-grade production, and regulatory approval ahead of future clinical trials.
Exploring New Treatments
Alongside gene therapy, researchers are developing mRNA therapies and nutrient-based approaches to support future treatment options.
Join Our Monthly Lottery
By joining the Joseph’s Goal Monthly Lottery, you’ll be doing something amazing. Every ticket supports life saving NKH research and helps families facing this devastating condition. And of course, you’ll be entered into our monthly draw for the chance to win.
A small contribution each month can make a huge difference.