We are living a parents worst nightmare. Our sweet son Darian, was recently diagnosed with a very rare and fatal 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 10 years to live. Currently there is no cure for GM1.
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 we are now searching far and wide for any support we can muster in our fight to save our son’s life.
There is some hope in all this sadness. There is an experimental treatment which could cure or pause the disease and is about to begin human trials in the coming months. But there are many if’s between our current reality and him getting treatment. There is no time to waste as Darian’s disease is progressive and has already begun taking a toll on his body. He can no longer walk independently but his smile is shining as bright as ever.
Our top priority is to get Darian into the upcoming trial. 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. What we’re looking for 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.
While our primary focus is the trial, 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.
We know we are not alone in this fight to save Darian and can’t thank everyone enough for all of the support.