Realizing the Dream Gene Therapy Program: Phase One

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$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


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.