DUCHENNE MUSCULAR DYSTROPHY
20% of sales will go to Kilian's family to aid in his journey.
Kilian’s journey began in January 2023 when he was diagnosed with Duchenne Muscular Dystrophy (DMD). DMD is a rare and progressive disease that affects the skeletal muscles. It is caused by a mutation of the gene that tells the body to produce dystrophin, the protein that our body needs to build muscle and strength.
Since receiving the diagnosis, Kilian has begun physical and occupational therapy to improve his gross and fine motor skills. In addition to therapy, Kilian was placed on intensive steroids to reduce inflammation which has helped to improve his overall mobility.
Currently, there is no cure for DMD. However, at the end of the summer, the FDA approved the first ever gene therapy (ELEVIDYS) for DMD. This gene therapy shows promise in stabilizing the progression of symptoms, and, in some individuals, improving strength and endurance at the ages when children with DMD usually become weaker. Kilian has been medically cleared by his doctors and his insurance has just recently approved the treatment. Kilian is scheduled to receive the one-time infusion at the end of October.
Kilian’s physical and medical needs will be ongoing for the rest of his life. As he grows older, there will be a need for an accessible mini van to assist in ease of transfers in and out for both Kilian and his parents. The additional cargo space will also be needed to transport his medical equipment, some of which will likely not be covered by insurance in the intervals that are needed as he physically grows. In addition, renovations to the house will also be needed in the future to make the house more accessible for him to get around.
This fundraiser seeks to assist the family in meeting some of these ongoing care needs.