$200K For SMA—Phase Two | $100,000 Granted | Funded SMA Large Animal Model Research

$200K For SMA—Phase Two | $100,000 Granted | Funded SMA Large Animal Model Research

Once again, with your incredible support we have built on the wave of momentum from the "$200K For SMA -- Phase One" campaign to place a material, targeted grant in the hands of a macro focused SMA research program. In just a matter of months since launching "$200K For SMA -- Phase Two" in parthership with FightSMA in December 2011, we awarded $100,000 to Dr. Monique A. Lorson and University of Missouri in support of Dr. Lorson's research focused on developing a large animal model of Spinal Muscular Atrophy (SMA).

Currently, no large animal model of SMA exists and the goal of Dr. Lorson's research is to develop the first ever pig-based model of SMA to be used by the broad, global SMA research community and other research groups to allow them to more efficiently and effectively move promising research from the bench to bedside. In short, this is a very exciting program that has the potential to have a material, positive impact on the future of SMA research in almost every category (e.g. gene therapy, compounds, antisense oligonucleotides).

A quote from Dr. Monique A. Lorson of University of Missouri:

"With the support of FightSMA and the Gwendolyn Strong Foundation, our collaborative team at the University of Missouri has made important progress towards developing a large animal model of SMA. Our goal is to generate two animal models of SMA in the pig: the first model specifically addresses therapeutics aimed at changing SMN2 splicing and the second reflects the true SMA disease model. The first animal model has been generated and we are preparing to evaluate delivery parameters and therapeutics aimed at increasing SMN2 exon 7 splicing in these pigs. We are also on our way towards having the SMA disease model. We are grateful to FightSMA and Gwendolyn Strong Foundation for their support of this project. This award will be used to generate SMA pigs and further develop the SMA animal model for therapeutic studies."

To say that Dr. Lorson's program has big potential is a massive understatement. In fact, researchers around the globe are excited about Dr. Lorson's SMA large animal model and how it may help answer important questions and streamline the research process. Dr. Lorson's program should provide a more effective model for these researchers to test their potential therapies and allow government entities, pharmaceutical companies, and nonprofit organizations like ours to have a higher potential return on their research funding investment as we continue to collectively work towards finding a potential therapy for SMA in the future.

Thank you for your continued support! It HAS made a difference.

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