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Making a Difference Through Science – A New Hope in the Fight Against Duchenne

A light shines through brightly and proudly, a light powerful enough to illuminate the darkest of obstacles. That light is hope—one of the hardest things in the world to extinguish. For those affected by Duchenne muscular dystrophy (DMD)—a new hope might be on the horizon, thanks to science. JAR of Hope, in collaboration with a leading research organization, is testing a drug that could provide a viable and effective solution for treating a disease that cuts short far too many young lives.

This drug, called JAR913, which was recently secured by JAR of Hope for testing, could potentially have a profound impact on slowing the progression of DMD. How so? The answer is simple—more dystrophin. DMD occurs when there is a mutation to the dystrophin gene, which impedes the body’s production of this crucial protein. The disease results in muscle weakness, the delayed ability to sit, stand, or walk, and difficulty in running and climbing stairs. DMD, which starts in the pelvic area, upper legs, and shoulders, eventually moves to the trunk, arms, and legs. Most children are reduced to a wheelchair by their teenage years and in many cases, succumb to the illness by their mid-twenties.

JAR913 aims to reduce symptoms of DMD by repairing the dystrophin gene, thus increasing the amount of dystrophin created in the body. Biomere, one of the nation’s premier clinical research organizations, is currently studying the drug. The study will test the drug on the following criteria:
Proof of Concept and Efficacy: Biomere’s stringent testing will determine if JAR913 is both feasible and practical for use on DMD patients. Following that determination, the testing organization will look to confirm the drug’s efficacy in treating the disease.

Safety and Toxicity: JAR of Hope and Biomere are both committed to upholding the highest of safety standards. In order to pass testing, JAR913 must prove 100% safe for use and nontoxic, especially considering most Duchenne patients are young children with already compromised immune systems.

JAR of Hope Founder and CEO, Jim Raffone, stands confident that the drug will be a huge breakthrough in the fight against DMD. “We have some of the nation’s brightest minds testing this compound, and we believe, from what we know about its properties, that it can slow down the progression of Duchenne. While nothing is ever assured, JAR913 could have a significant effect on the Duchenne community as a whole and bring us closer to a cure.”

The trial period for JAR913 began in November 2018, with Biomere estimated to release its findings in the first quarter of 2019.

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