Mizzou Give Direct

College of Veterinary Medicine

Goal: $300,000

$202,991 (67%) raised Need: $97,009

College of Veterinary Medicine

Fund to Cure SMARD

Silas Werner and Catherine Sims both baffled small armies of pediatric specialists before each was diagnosed with SMARD. SMARD is a rare, life-threatening motor neuron disorder that affects infants and children. During agonizing hospital stays as infants, Silas was diagnosed in 2010 at 3 months and Catherine was diagnosed in December 2013 at 4 months. Both sets of parents, Lisa and John Werner, and Jill and Eric Sims, were devastated. 
Silas and Catherine -- nearly 4 years apart in age and diagnoses and living in two different states -- didn’t know about each other, although their paths were uncannily similar. The horrifying news of both Silas and Catherine's SMARD diagnoses came after the deaths of Silas’ older sister, Valentia, and Catherine’s older brother, Bobby. Valentia and Bobby’s deaths were listed as SIDS (Sudden Infant Death Syndrome), despite the absence of risk factors. When Catherine and Silas were later diagnosed with SMARD, it was confirmed that Valentia and Bobby also had SMARD. 
The Werners' and the Sims’ paths finally crossed when Jill Sims was searching for a diagnosis for Catherine and stumbled across Silas’ story online. The two families have since been working together brainstorming on how to cure SMARD. Living against all odds, Silas and Catherine both breathe with a ventilator and require round-the-clock care supplied by visiting nurses and their parents. Despite their conditions, Silas and Catherine radiate joy from their illuminating smiles and are happy children. 
The Werner and the Sims families are part of a close network of SMARD parents who now have newfound hope for the future. Christian Lorson, PhD and Monir Shababi, PhD have recently published results demonstrating that early delivery of a gene therapy vector to SMARD-model mice significantly extends survival. The Lorson and Shababi lab at the University of Missouri is continuing to pursue the development of a gene therapy approach to replace the mutant gene in SMARD. Since this gene is defective in all SMARD patients, gene therapy may be a therapeutic approach for newly diagnosed as well as older SMARD patients. 
Hidden behind the glowing smiles of these two beautiful children is an urgent need for a cure or treatment for this devastating disorder. Other children across the globe have the same need. As of date, there is no federal funding for SMARD research. Through a generous donation from the Dr. Grant Sims and Mrs. Patricia Sims, Dr. Lorson and Dr. Shababi will examine a promising therapeutic approach in older SMARD-model mice. “We are very grateful for the support from the Sims family and are extremely excited about the opportunity to continue to push this potential therapeutic closer to the clinic,” Lorson said. “Molecular medicine and gene therapy are entering a new and exciting phase of discovery and we hope that SMARD can benefit from this type of therapy in the future.

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