Help Us Raise “200K for SMA” Gene Therapy Research!July 06, 2010 by Victoria Strong
$200K for SMA --> One person and one dollar at a time. Together we can end SMA!Why Your Support Matters -->
The Gwendolyn Strong Foundation (GSF) is a 501(c)(3) nonprofit public charity dedicated to ending Spinal Muscular Atrophy (SMA), the leading genetic killer of infants and young children. As witnesses to the degenerative cruelty of SMA, it is our passionate mission to accelerate research solely focused on curing this devastating disease. GSF created the $200K for SMA campaign to direct 100% of your generous donation to fund groundbreaking gene therapy research with the potential to treat SMA forever. Weve partnered with FightSMA, who has a long-standing stake in gene therapy, to help make a decade-old dream come true. Your donation is providing hope for thousands of families and is an investment in a future where SMA is no longer deadly.
Why Gene Therapy -->SMA is an ideal candidate to benefit from gene therapy because the SMA genetics are very clear and well understood. A single gene is responsible for all clinical forms of the disease and gene therapy is a technique with the potential to treat SMA by inserting a corrected form of this single gene into a patients cells instead of using drugs or surgery. Our SMA gene therapy program at Nationwide Childrens Hospital and The Ohio State University continues to show great promise for treating SMA patients, said Dr. Brian Kaspar. With this award, we will be able to test our therapy for potential safety or toxicity issues and provide important information for the regulatory process as we advance our program to the clinic in a swift and safe manner."
Why $200K -->GSF has committed to raising $200,000 for FightSMAs gene therapy program to help bring this promising research to clinical trial. With a grant from GSF and FightSMA, Dr. Kaspar has already begun work to prove safety and toxicity of this research, but in order to take gene therapy out of the lab and into treatment, much more work must be done. We are determined to make sure funding is not an obstacle in moving those studies forward. With an additional $200,000, Dr. Kaspar and his team can complete their current studies and begin moving on to other studies required to submit for FDA approval. $200,000 will by no means fund the entire project, but with a reputation that has earned him trust and recognition, the completion of these initial studies are also critical to allowing Dr. Kaspar to garner additional funding from government and other sources. Your donation is an investment in continuing this important relay race and helping bring this research to those impacted by this cruel disease.
Why Partner -->By combining efforts and strengths, it is our hope that this exciting research will be funded more efficiently. By coupling FightSMAs strong SMA research history, relationship with gene therapy researchers, and their esteemed team of scientific advisors with GSFs history of bringing people together and creating successful and innovative campaigns, collaboration seemed the natural next step for this program. And together FightSMA and GSF have already awarded Dr. Kaspar $175,000 for his program and this additional $200,000 is part of a long-term commitment to this promising research.
How Do I Get Involved -->Simple. Anything can become a fundraiser. Get creative. Think outside the box. And then go for it. All that we ask is that you:
- Set a goal and strive to meet it.
- Plan to have all funds raised by the end of 2010.
- Have fun!
100% of your donation to the Gwendolyn Strong Foundation under the $200K for SMA campaign will go directly to this exciting research. Thank you for making a difference!!!
$200K for SMA --> One person and one dollar at a time. Together we can end SMA!