Hope for 'Butterfly' Kids Thanks to New Treatment
Their rare genetic disorder can cause severe blistering all over the body
By Elizabeth Armstrong Moore, Newser Staff
Posted Aug 8, 2014 9:59 AM CDT
Image of bone marrow.   (Shutterstock)

(Newser) – When Elisa McCann was born 18 months ago, her parents knew something was wrong. Their third child had blisters everywhere on her body within her first day of life, and doctors quickly diagnosed a rare genetic disorder called epidermolysis bullosa (EB), reports Today. In kids with the condition, who are sometimes called "butterfly children" for having skin as sensitive as butterfly wings, there is a defect in the gene that makes collagen, so their layers of skin don't adhere together properly. Any external trauma, even minor bumps and scrapes, can cause friction that can lead to blistering.

Fortunately for Elisa, her symptoms have been improving dramatically since she became the 28th child to enroll in an experimental stem cell trial at the University of Minnesota where kids with EB get bone marrow from healthy donors (Elisa's older sister is her donor). The procedure introduces healthy cells to their blood that help reverse the disorder, notes Today. But the estimated 25,000 to 50,000 people in the US with EB—mostly children—who haven't undergone the treatment face uphill battles, and some families must grapple not only with the disease but also with $2,000 monthly bandage bills, reports First Coast News. (NIH's new $43 million quest: Study the diseases so rare they often affect only 50 people on the planet.)

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Showing 3 of 4 comments
JCinVA
Feb 3, 2016 7:39 AM CST
The one's who die young have the most severe type, which is what this article is discussing. There are many types of EB, my husband and children have the least severe type, which is manageable. They get painful blisters, but primarily on their hands and feet, and not at a touch, but repeated friction (e.g. crawling, running, jumping, swinging (hands), climbing across monkey bars, etc.). It has gotten a little better as my husband has gotten older, mainly because his feet are callused compared to children's soft feet. To learn more about Epidermolysis Bullosa I recommend you go to www.debra.org. Please consider donating to EB research and charities, they are always in need because funding for rare diseases is not as plentiful as heart disease, cancer, etc.
ms_tiff86
Aug 8, 2014 4:23 PM CDT
Unfortunately, i think they do not live past childhood. There was a child with EB close to our hometown, his mother was an RN and she kept a blog throughout his fight with this disease. He passed away at age 2 1/2. Very heartbreaking story. Here is her blog address: http://randycourtneytripproth.blogspot.com/
crafter67
Aug 8, 2014 2:27 PM CDT
Mostly children...Do they grown out of it naturally? what a horrible, painful sounding disease.