$200K For SMA—Phase One | $250,000 Granted | Funded SMA Gene Therapy Research
Goal set, goal met, goal exceeded. Once AGAIN! Six months. Thousands of donors. Hundreds of organizers. 60+ fundraisers. Together we not only raised $200,000 for promising SMA gene therapy research. We raised $250,000+! That's inspiring! That's awesome! And together we have once again made a difference in moving promising SMA research forward.
In July 2010, GSF created the "$200K For SMA -- Phase One" campaign to fund groundbreaking gene therapy research at Nationwide Children's Hospital and The Ohio State University led by Dr. Brian Kaspar. We partnered with FightSMA , who has a long standing stake in gene therapy, to help make a decade-old dream come true. In just six months after launching the fundraising challenge, the Gwendolyn Strong Foundation — and all of our wonderful supporters — not only raised $200,000 for gene therapy research — we all blew it out of the water! With the help of literally thousands of donors, hundreds of organizers, 60+ fundraisers, together we raised more than $250,000! In. Just. Six. Months. And because we raised beyond what was expected, we were able to begin funding the next critical phase (see our "$200K for SMA -- Phase Two" campaign) of necessary research required to bring gene therapy out of the lab and to the thousands impacted by SMA!
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 patient’s cells. Dr. Kaspar's research was published in the Nature Biotechnology.
So, what has been funded with this $250,000? >> Critical Safety and Toxicity Studies for the SMA gene therapy program led by Dr. Brian Kaspar at Nationwide Children's Hospital and The Ohio State University.