As you may have heard, my niece’s son, Darian, was recently diagnosed with a very rare and terminal genetic disease called Gm1 Gangliosidosis. In short, his body is not producing a protein which is essential for the function of his nervous system, giving him less than 5 years to live, he just turned 2 years old.
This is that moment in life you wish never to occur, not for oneself nor for any other human being on this planet. But unfortunately this is happening and I am now searching far and wide for any support I can muster in our fight to save his life.
There is some hope in all this sadness. There is an experimental treatment which could cure him and is about to begin human trials in the coming months. But there are many if’s between our current reality and him being cured. There is no time to waste as Darian’s disease is progressive and has already begun taking a toll on his body.
To that end I am reaching out to you for support in the any and all of the following ways…
The Cure – This is first and foremost our priority right now. The pharmaceutical company Axovant in conjunction with the NIH are awaiting FDA approval to begin human clinical trials for a gene therapy which has shown promising results in curing the disease. What we’re looking for here is anyone who could possibly help us get this trial approved faster by the FDA and also to ensure Darian is selected for the trial.
Further Contacts – While our primary focus is the trial above, we are looking far and wide for any other research centers, pharmaceutical companies or medical professionals who might also know of anything or are working on anything that could help save Darian’s life.
Crispr – we know that this is a nascent field but any information or contacts who are involved in this field to help us better understand it would be greatly appreciated. It’s a long shot but we’re looking for all possibilities.
Alternative Medicines – Finally, though we are putting our trust and energy behind the scientific community on this one, we will of course look into anything that will save him, including any alternative medicines. So please do send anything you think might help.
If you know anyone else that could help us on this journey please do share this with them.
You know me, I am not someone who would call upon you in this way unless I thought it absolutely necessary. So please if there is any way you could help us save Darian’s life, please please do it. You can reach us via email at: firstname.lastname@example.org
(A photo of Darian from 2018)