The main NKH Awareness Day fundraiser is on facebook, if that is more convenient for you:  https://www.facebook.com/donate/303945447916408/

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May 2nd is NKH Awareness Day - we're asking instead of your daily flat white, please swap your coffee today for a donation towards NKH Research.

Please. Please donate. £3. £5. £15. Whatever you are able.

There is an honest-to-goodness CURE in the pipeline, and all that stands between that and our kids is money. Please please donate.

If donations aren’t your thing, please buy an Eva the Adventurer book on Amazon (all royalties go to Joseph’s Goal). Please change your Amazon Smile charity of choice to Joseph’s Goal. Please change your profile picture on facebook to the NKH frame. Please share this fundraiser.

Here’s what I can tell you. Your funds are making a difference. In the last year, there has: 

- New published papers (including understanding the GCSH gene’s affect on NKH) 

- Better understanding of how NKH works. Specifically, at how glycine metabolism in the brain/liver affects neuromuscular function, and how NKH changes specific brain structures, across the three different mice models. They’re also looking at the biochemical and molecular markers of liver and neural cell models with NKH, and how they differ to liver and neural cell models without NKH. 

- Gene Replacement therapy (aiming to place a working copy of the GLDC gene into the body) is moving forward, and have selected a vector to express human GLDC, and to treat mice. The initial levels show in the treated mice the new GLDC gene is working (!!!), and further mice are being treated to expand the study, so they can test for the correction of metabolic and neurological function. In parallel they are long-term safety testing. 

- A new treatment with sodium cinnamate could be an alternative to Sodium Benzoate, and preclinical work is being done with the regulatory authorities as a precursor to clinical trials.

You GUYS! Every donation is enabling real research! You are paying for real research, real research that is underfunded, real research that has the potential to improve children’s lives dramatically. Our children's lives!

So please donate today. Please go out and tell someone about NKH. Tell them about Mikaere or Joe or the baby you know with NKH.

Organise a fundraiser in your office, a bake off, a poker match, a virtual fun run. The stakes are small, and every single donation helps. Every single donation is funding research that will change lives.

Happy NKH Awareness Day. Thank you for being in our corner. We love you.

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Joseph's Goal is UK's only NKH Charity, who funds research by Prof. Nick Greene and his team at UCL. You can read more about their work here: https://www.ucl.ac.uk/child-health/research/developmental-biology-and-cancer-programme/developmental-biology-birth-defects/andrew-5 More about Joseph's Goal at josephsgoal.org

Donating through JustGiving is simple, fast and totally secure. Your details are safe with JustGiving - they'll never sell them on or send unwanted emails. Once you donate, they'll send your money directly to the charity. So it's the most efficient way to donate - saving time and cutting costs for the charity.