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4-year-old who was deemed 'lazy' finds hope in new drug that treats rare disease
Fox ^ | 1-18-17

Posted on 01/18/2017 7:10:23 AM PST by workerbee

A new drug is offering hope for the family of a little girl whose genetic defect causes mobility issues similar to that of polio and amyotrophic lateral sclerosis (ALS).

Four-year-old Lexi Pacini, of Colorado Springs, was diagnosed with type 2 spinal muscular atrophy (SMA) as a toddler. The rare disease is marked by the absence of a gene that codes protein signals from motor neurons to muscles, and it leads muscles to weaken to the point of atrophy.

**SNIP**

“She dances the way she knows how, in her walker,” Tammy told Children’s Hospital Colorado in the release. “She just doesn’t give up. She wonders why she can’t do certain things, and we’ll tell her, ‘Well, you’re just not as strong. You don’t have as much muscle.’ And she’ll just say, ‘No, I have muscles. I’m strong.’”

(Excerpt) Read more at foxnews.com ...


TOPICS: Health/Medicine; Miscellaneous
KEYWORDS: drugtherapy; raredisease
Just a bit of good news for a change. A reminder to thank God every day for the roads we don't have to walk.
1 posted on 01/18/2017 7:10:23 AM PST by workerbee
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To: workerbee

How can this be? I though the pharma industry was poisoning us, or trying to make us all into autistic robots? </s>


2 posted on 01/18/2017 7:16:01 AM PST by oblomov (We have passed the point where "law," properly speaking, has any further application. - C. Thomas)
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To: workerbee
Almost as good as this Onion article: Brain-Dead Teen, Only Capable Of Rolling Eyes And Texting, To Be Euthanized

The parents of 13-year old Caitlin Teagart have decided to end her life, saying she can now do nothing but lay on the couch and whine about things being "gay."

3 posted on 01/18/2017 8:42:17 AM PST by DCBryan1 (No realli, moose bytes can be quite nasti!)
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