NKH Research
Advancing NKH Research Together
For more than 14 years, Joseph’s Goal has been helping to drive research into Non-Ketotic Hyperglycinemia (NKH), supporting the work of Professor Nick Greene and his team at University College London (UCL).
Since the charity was founded in 2012, our supporters have helped us raise an incredible £1.3 million for NKH research.
What began with one family determined to create hope following Joseph’s diagnosis has grown into a long-term commitment to advancing our understanding of NKH, developing potential treatments and helping researchers move closer to changing the future for children and families affected by this ultra-rare condition.
We are incredibly proud of how far the research has come – and even more excited about what comes next.
Our fundraising has also helped support the work of Professor Johan Van Hove and his team at the University of Colorado, making Joseph’s Goal part of an international effort to better understand NKH and develop new treatments.
None of this would be possible without the people who have fundraised, donated and supported Joseph’s Goal over the years.
Together, we are making a real difference. 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.