The Gwendolyn Strong Foundation (theGSF) and FightSMA, are excited to announce a $100,000 award 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).

SMA is the leading genetic killer of young children, occurring in approximately 1 in every 6,000 live births and with a genetic carrier frequency of approximately 1 in 40. However, no treatment or cure exists. Currently, researchers around the globe are working on many fronts to develop effective SMA therapies using the available SMA mouse models. However, it is widely known that these mouse models have significant limitations. It is anticipated that since the pig has many biological and physiological similarities to the human that an SMA pig model will more closely mimic the human condition and, therefore, provide researchers with a more realistic model through which to test potential SMA therapies.

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.”

“We are unbelievably honored to announce this targeted $100,000 research grant,” said Bill Strong, theGSF co-founder. “This is an exciting program; with its potential big-picture impact and long-term benefits to SMA research across the board. 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.”

“This research initiative is a game-changer,” said Martha Slay, founder of FightSMA  “We’re excited about the potential that this model has for the full spectrum of SMA research and the impact of this research on our community.  We are equally excited and honored to continue our collaboration with the Gwendolyn Strong Foundation.”