Curing Caroline: A GrandFather's Fight To Save The Life Of His Granddaughter

- A well known Houston pediatric neurosurgeon is trying to save the life of his first grandchild born with a rare disease.  Finding a cure for her is not easy, but it could mean help for thousands of others living with similar, incurable diseases.   

As babies, Caroline was crawling faster than her twin brother Henry.

"They were progressing the same, and at about 9 months we noticed a regression in crawling," said Dr. Stephen Fletcher.

Caroline's rare genetic mutation, similar to something called Charcot Marie Tooth Disease, causes weakness in all her muscles.  She can't walk or use her hands.  Her immune system is easily compromised.  Caroline's aunt started the facebook page, "Curing Caroline," hoping a combination of science and social networking will lead to a cure.

The family was able to connect with scientists at the world famous Jackson Laboratory.  Dr. Fletcher says mice with the same genetic mutation as Caroline have recently shown progress with gene therapy.  They're trying to fund an expensive treatment that, if successful on Caroline, could help thousands of others who suffer from neuromuscular diseases like ALS.

For now, they're focused on building up her strength with the hope she'll be walking again soon.



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