Their primary focus is repurposing amlexanox, an anti-inflammatory drug that has been used for a variety of conditions, including asthma and diabetes. With a proven safety profile and low production costs, amlexanox has shown success in preclinical studies for genetic disorders like cystic fibrosis, Duchenne muscular dystrophy, Charcot-Marie-Tooth disease, Aspartylglucosaminuria, and epidermolysis bullosa; in order to bring amlexanox back to market, they are working towards FDA approval with orphan drug designation to ensure this treatment is available to the rare disease community at an affordable cost.