Realizing the Dream Gene Therapy Program: Phase One

>>Click here to read about Phase Two of the Realizing the Dream Gene Therapy Program

$250,000 Goal Reached and Exceeded!

FightSMA and the Gwendolyn Strong Foundation are thrilled to announce that we have reached and exceeded the $250,000 goal set out for our Realizing the Dream Gene Therapy Program. The money was raised in just six months! To say we’re thrilled doesn’t describe the emotion.

From the Gwendolyn Strong Foundation’s Victoria Strong:

With the help of literally thousands of donors, hundreds of organizers, 60+ fundraisers, and through our partnership with FightSMA, together we have raised more than $250,000!!! In. Just. Six. Months. And because we have raised beyond what was expected, we are able to already begin funding the next critical phase of necessary research required to bring gene therapy out of the lab and to the thousands impacted by spinal muscular atrophy!!!

From FightSMA’s Science Director, Dr. Chris Lorson, PhD:

This would not have been possible without the extremely dedicated effort from the Strong family and all of the SMA families and friends that reached out to help over the past several months. The gene therapy project is led by Dr. Brian Kaspar (Nationwide Children’s Hospital). Recently, Dr. Kaspar and colleagues have shown dramatic survival benefits using scAAV9-SMN vectors. It is a long road from mice to humans and to bring this vector closer to a clinical trial, many steps must occur, with safety and toxicology studies being essential. This is where FightSMA and The Gwendolyn Strong Foundation have come together and provided support. This is clearly a community-wide effort and it will take more money and more time to bring a gene therapy trial to fruition and FightSMA and The Gwendolyn Strong Foundation are continuing to push hard to make this a reality.

From Dr. Brian Kaspar, PhD, Principal Investigator at Nationwide Children’s Hospital, whose lab has received funding for safety and toxicity through this campaign:

I am very thankful for the FightSMA and Gwendolyn Strong Foundation funding of $250,000 our lab has received to date — funding that has allowed the initial safety and toxicity studies to be conducted. We are in the middle of these key studies, and so far, all looks good.

So, who is responsible for this great accomplishment? You are! FightSMA could not have been successful without help.

FightSMA Chapters and Partners

Gwendolyn Strong Foundation

Andrew’s Buddies of the

Tri-State Area

Andrew’s Buddies of New York

Andrew’s Buddies of Illinois

Corinna’s Angels/FightSMA Rhode Island

Desiree’s Buddies/FightSMA Southwest

Andrew’s Buddies/FightSMA MechanicsvilleC

Dylan’s Friends/FightSMA Utah

FightSMA Alabama

FightSMA Connecticut

Morgan’s Buddies/FightSMA Tidewater

FightSMA Richmond

Hannah’s Buddies/FightSMA Tampa Bay

Joshua’s Buddies

FightSMA Nevada

Patrick’s Buddies

Shannon’s Buddies

SMAtlanta/FightSMA Atlanta

Tori’s Buddies/FightSMA Canada

StopSMA

The Wallace Family

Grey’s Gang/Charlotte, NC

Climb4SMA/Jadon’s Hope

The Kennedy Family 

Michelle Nyhuis 

Thanks to everyone who made this possible!

Note: Article taken directly from FightSMA, click here for the link.

SMA BLOG PARTY: This is SMA

Brielle and Brooke, both have Spinal Muscular Atrophy, type II. SMA is a devastating disease and something I was not familiar with before Brielle’s diagnosis. Since that diagnosis, our lives have changed so much. So many hopes and dreams for our kids went out the window. We still have so much hope for research to find a treatment/cure for this cruel disease and we do our best to have a positive outlook on life and do as many fulfilling experiences for our girls as we can. Our friends and family have been with us every step of the way and we couldn’t have made it through those really tough times without them.



…and even our friends of friends want to help. The owners of the restaurant “Rusty Rudder” on Lake Norman in North Carolina are friends of my sister (aka Aunt Christen) and they held a golf tournament last Monday and want to give all the proceeds to Families of SMA in honor of Brielle and Brooke. They held a golf tournament at Skybrook golf course, then complimentary food and live music and a silent auction at the Rusty Rudder after. The music was awesome and they even let Brielle play the drums along with the guys playing the live music. It was a perfect night and a beautiful sunset. A night we will never forget and we can’t thank them enough for choosing SMA for the proceeds!

Skybrook Golf Course, North Carolina

Golf Cart Rides

Golfers getting ready to start

mom, brooke, grandma, brielle, aunt christen, great grandma (daddy had to stay home to work).

It was warm…

This hole sponsored by HMS.

Trying to get a “hole in one” to win 2 brand new jet ski’s.

Expert golfer, C. O’neal  ;)

Complimentary sponsorship by Friends of Brielle & Brooke

Brielle playing the drums at the Rusty Rudder later that night.

Beautiful sunset over Lake Norman

What is SMA?

SMA kills more young children than any other inherited disease, is the most common cause of genetic infant mortality, is the #1 genetic killer of young children.

SMA is degenerative, causing the weakness and wasting of voluntary muscles — eventually impacting crawling, walking, standing, sitting up, coughing, breathing, swallowing, speaking…anything requiring muscle strength.

SMA is estimated to occur in 1 out of every 6,000 live births. Incidence rates are comparable to the more well known ALS/Lou Gehrig’s disease and Cystic Fibrosis.

1 in every 40 people unknowingly carries the SMA causing gene or nearly 8 Million potential parents in the U.S. alone — making it “common.” Both people must be carriers for the disease to be passed on — making it “rare.” There are simple blood or saliva tests available to learn if you are a carrier before you get pregnant. The American College of Medical Genetics recommends ALL couples be tested for SMA.

SMA is terminal. There is currently no treatment or cure… BUT there is much research work being done around the world and it holds enormous potential. In fact, the National Institutes of Health selected SMA as the disease closest to treatment.

SMA research is drastically underfunded –> YOU can help!

PLEASE VOTE: It’s really this simple –>

You can vote once EVERY DAY from now until September 29th at 5 PM EDT

Go to VoteForSMA.com, select the Gwendolyn Strong Foundation, enter the two security words, check the Official Rules box, and then click “Vote Now!”

That’s it! It’s really that simple

Don’t forget to add your name to the DailyVoteReminder.com list so you don’t miss a $20K vote.

Brielle and Brooke with Chad Knaus, crew chief of #48, Jimmie Johnson!

Helmet of Hope sign in the 24/48 shop at Hendrick Motorsports