Long story short: my daughter is 5 and my son is 3, they both have been diagnosed with a rare, terminal disease called Infantile Neuroaxonal Dystrophy (INAD). This disease has already taken so much from them, they can’t walk, talk, eat, sit or even laugh. But there’s a new drug that has been helping. The kids are part of a clinical trial for a new drug, but it's in NJ and is very expensive to go back and forth to the trial site from KY. The new drug has shown to be beneficial, Riley has been smiling more often and moving her arms, and Harrison has been able to move more and is getting more vocal.
Here’s the whole story:
Riley is 5 years old and her brother, Harrison, is 3. They were both diagnosed with a very rare, terminal disease called Infantile Neuroaxonal Dystrophy. (Also called INAD) INAD is so rare there are less than 100 children in the entire world who have been diagnosed. The prognosis is grim, with life expectancy of 10-12 years old with a very poor quality of life. Children with INAD start out as normal children for the first year, learning to talk, crawl and sometimes even walk. But then they slowly start losing all their abilities. They lose their balance, they stop walking, talking, crawling and sitting. Then they lose control of their body and stop being able to move altogether. They are unable to eat, drink, and even urinate without medical assistance. Children with INAD usually suffer from seizures, deafness and blindness, but Riley & Harrison are not dealing with those symptoms right now, although they both suffer from painful muscle spasms and are on medication for nerve pain.
INAD allows membranes to form over the nerves, which blocks messages to and from the brain, basically trapping the child in a body that refuses to work. Eventually the children lose all muscle tone, which makes it difficult to cough or laugh. Being unable to clear their lungs, children with INAD are extremely susceptible to respiratory infections, and most children with INAD die from respiratory failure.
Riley and Harrison are both floppy and fragile as newborns and have had many medical issues. They both have feeding tubes and are unable to speak or even hold their own heads up. Although Riley & Harrison are suffering from a very terrible disease, they are both such happy children. Harrison is always laughing and smiling and loves meeting new people. Riley loves adventure and going new places and is especially fond of drama (she enjoys Hell’s Kitchen and Jerry Springer). They are both such amazing, strong and resilient children. Even though the prognosis is grim, there is hope. There is a clinical trial for a new drug that can possibly stop the progression of the disease and even reverse some of the symptoms. Riley and Harrison have been in the clinical trial since December 2018 and have shown some improvements. But the clinical trial is in New Jersey and it’s very expensive to drive 16 hours one way and stay in a hotel for a week at a time during the treatment. This year our goal is to raise money so we can not only afford to continue this life-saving clinical trial, but also help cover other expenses. With 2 medically fragile children, it’s impossible to have both parents working outside the home and living on one salary has been extremely difficult. But we are willing to do whatever we need to do to help our children and make sure they have the best life possible.