NZ Family Races Against the Clock to Fundraise for Son’s Rare Disease Treatment

Nash Jones is 18 months old and he is battling against the clock for a better quality of life.

Nash Jones has a very rare genetic disease called Lesch-Nyhan syndrome. This disease affects young boys, causing intellectual disabilities, uncontrollable muscular movements, kidney issues, and self-injury. The self-inury (which affects roughly 85% of people with the disease) consists mainly of biting and head banging. Patients may bite their fingers, toes, legs, lips, etc. Many people with this syndrome only live to be about 19 or 20, due to kidney failure.

Nash’s parents, Fran and Nathan, are interested in a stem cell transplant for their son, which could prevent self-injury behavior. Nash, who was diagnosed at 9 months of age, has not started self-injuring. Thisy aspect of the disorder usually occurs between 2 and 3 years of age. Nash is eighteen months old.

Besides the stem cell transplant, the family’s other option would be to remove Nash’s teeth, put him in head gear, and strap him down at night to keep him from hurting himself at night.

The stem cell transplant is very new. It’s still experimental in New Zealand, where the family lives. To get the treatment, the family would come to the US, where they would live for up to a year, footing the cost of living and other expenses.

The price tag for the operation rests at about 2.5 million dollars.

Nathan says if their participating in the new stem cell transplant can potentially help a family like theirs, wrestling with this diagnosis, in the future, “it’s 100% worth it.”

The situation is urgent. Nash’s parents feel like they are playing “mission impossible,”– they are in a race against time and need to act quickly before Nash’s self-injury behaviors begin. The stem cell transplant has a much lower chance of being successful if these behaviors have already started. It’s important to note, is that the transplant only stops the self-mutilating behaviors; Nash’s other complications will not be improved by the treatment.

So far, the family has raised over $43,000 from Givealittle, which is only a small fraction of the total cost of the procedure.

Nash’s mother Fran says if everyone in New Zealand gave five dollars– roughly the cost of one cup of coffee– they would be at goal.

Read original article here.

 


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