$200K For SMA—Phase One | $250,000 Granted | Funded SMA Gene Therapy Research

$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.

Related Blog Posts



Talk About It