Hollywood celebrities such as Rihanna and Jessica Alba rally behind a family who is currently fighting a devastating disease affecting their two little girls.
This disease is known as late infantile NCL or Batten disease. An inherited neurological disorder, it is caused by a defective gene called CLN6, which causes children to suffer from seizures, blindness, and ultimately muscle coordination loss. It is progressive, and children usually don't live past their twelfth year.
This is the same disease that is now afflicting the only two children of Kristen and Gordon Gray, a movie producer in Hollywood. The symptoms were gradual, according to the mother, and began when the eldest, Charlotte, was almost 3 years old. She was repeating words until it seemed she could no longer learn anything new. Kristen noticed tremors in the arms when she tried to get up.
It took a while for little Charlotte to be diagnosed correctly since the disease is rare. But when the parents finally got hold of the testing, they also let the second one, Gwenyth go through it. The result was positive: she had the same gene.
Because of its rarity, there's no available treatment, although they have heard that some medical researchers are already learning more about it.
In the hopes they could find the treatment that can significantly delay the progression or even eliminate the disease, the Grays are now raising $12 million for the girls and for every child who's been diagnosed with Batten.
They have currently partnered with a team from Sanford, who's working with the Grays and another family in the same situation living in Israel. The research entails reproducing cells of the girls derived from the blood samples then identifying any of the existing drugs that may have the potency to reverse cellular damage caused by the disease.
In Batten, time is certainly of the essence. The family therefore hopes they can find the miracle they're looking for and receive good news as soon as possible.
