Josephs Goal - NKH Research

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.

Commitment that we recently took through the ONE MILLION POUND mark. In fact, £1.25 million pounds has been raised for NKH research from our little Joseph’s Goal charity.

We are incredibly excited at the work being done and the progress made towards NKH research.

Dr Greene recently presented us with a report as to the current state of his work… which, as he notes, is ‘thanks to the funds provided by Joseph’s Goal‘.

That tells us everything we need to know. That the years of hard work and toil behind the scenes is making real strides towards Joseph’s Goal – our goal – of eradicating this horrible disorder.

Finally, thanks to every single person who’s stepped up and helped Joseph’s Goal to fund the great work of Dr Van Hove in Colorado and now Dr Greene in London.

Together, we are making a real difference in the quest for a cure for NKH research.

It’s what our kids deserve.

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.