2 Before 2! (Cure L-CMD)

2 Before 2! ($2,000,000 before Austin's 2nd Birthday 8/27/21) raising money for rare disease gene therapy research.

$1,134,697

2.2k supporters

$2,000,000 goal

Team Member

Lee Souto

$250

by 1 supporters

Organizer

Created By

L-CMD Research Foundation

EIN: 85-2640499

Story

Please help us save our son’s life. Austin is 1.5 years old and has a fatal, muscle-wasting disease called LMNA-related congenital muscular dystrophy (L-CMD). In order to save Austin and similarly situated children, we have started the L-CMD Research Foundation. The purpose of the foundation is to fund the work of researchers and scientists who will develop treatments and a cure for L-CMD. The most pressing project, which could literally save Austin from childhood death, is the development of a gene therapy. The effectiveness of gene therapy decreases as children grow and as the disease progresses; so time is running out. We need to raise $2,000,000 to fund this gene therapy project. Our goal is to have this money by August 27, 2021—Austin’s second birthday. We are rawly and openly asking for your support of this project, which is our son’s best hope for survival.

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L-CMD is severe. Kids statistically live less than 18 years and succumb to respiratory insufficiency or heart issues. This progressive disease greatly affects Austin’s strength already. He has no head control, a weak torso, arms and legs, so he cannot stand, walk or even sit up or eat by mouth. We are currently discussing which power wheelchair will best suit him for his 2nd birthday.

Despite his limitations, however, Austin is quite happy. He loves music, "dancing," and bouncing in his baby bouncing chair (now much too small). He can't lift heavy toys or big books, but he likes to stack light blocks and play with any toy that makes a noise with buttons. So in these ways, he is cognitively quite typical for a child his age.

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Given that Austin is on the severe end of the spectrum, we cannot wait for the science to reach us; we must go seeking the innovative, most promising scientific technologies. Since his diagnosis in February 2020, we've been knocking on doors (virtually), talking with scientists and doctors, biotech firms and academic labs, and we've concluded that gene therapy will give Austin and the other kids with L-CMD the best chance to survive and thrive.

We know that the sooner kids with degenerative diseases receive gene therapy, the better the outcomes. The implications of this science could change the world, and we stand poised at the tipping point of making these treatments and cures a reality for many.

We KNOW that we cannot do this alone. We need your help.

HOW YOU CAN HELP TODAY:
1) Donate what you can
2) Share this fundraiser with your networks via email and social media
3) Share our social media posts
IG: www.instagram.com/lcmd.foundation
Twitter: https://twitter.com/FoundationLcmd
Facebook: https://www.facebook.com/LCMD.foundation
4) Reach out to us at [email protected] to discuss large donations, donating via check, organizing a fundraiser, company matching, corporate sponsorship, media/PR, grants


It ALL helps, and it’s ALL important.


WHERE IS YOUR MONEY GOING?
All money raised will go to develop and deploy a gene therapy being undertaken at a large academic research institution. We are moving with URGENCY to raise the money to make this happen.


WHY SO MUCH MONEY?

Research and development of cutting edge technologies unfortunately often falls to patient groups to fund them until such a stage that a large pharmaceutical company may see the monetary value and take it the rest of the way to full clinical trial and distribution.


URGENCY IS THE NAME OF THE GAME
Being a progressive disease, L-CMD will strip away Austin’s physical abilities to the point that he will no longer be able to move and his heart will fail to function properly without assistance. The sooner we can intervene, the better chance he will have to regain lost muscle function and stave off heart issues.


THANK YOU for considering being a huge part of Austin's success story. Let's change the world together because of the generosity of you, who see the light and joy in Austin. We are so hopeful.


We're ready. Let's GO!

THANK YOU FROM THE BOTTOM OF OUR HEARTS, Mark, Hannah, Ean + AUSTIN

L-CMD Research Foundation
EIN: 85-2640499 (501c3)
*All funds raised will be used for scientific research and development*

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$1,134,697

2.2k supporters

$2,000,000 goal

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L-CMD Research Foundation

A 501(c)(3) organization

EIN 85-2640499