If you told someone your child had cancer, they would immediately have a sense of the disease and potentially even a sense of the treatment options. People know about chemo, radiation and surgery. They don’t know much about rare diseases like Duchenne muscular dystrophy, and they are less likely to know that there is no cure and treatments are just beginning to become available.
Such is the experience for rare diseases, and the need for Rare Disease Day, always held on the last day of February. With only 15,000 boys affected in the US, Duchenne is one of those rare diseases.
The world is more focused on rare diseases now than at any time in the past. For the Duchenne community, this is a considerable blessing, adding support and interest for our research to find effective therapies and ultimately a cure for our boys. This past year has been a victorious one in many ways, with the approval of the first ever drug to treat the specific symptoms of Duchenne; though Exondys51 is only effective for 13% of the Duchenne population, it’s a big step in the right direction. Another drug EMFLAZA, a steroid, was also approved for all boys with Duchenne.
We have learned so much and yet still have so far to go. Other organizations have taken notice of our efforts, our business model in partnering with pharmaceutical companies to co-fund research and share the rewards, and our results. It’s exciting to see the community of rare diseases sharing knowledge and increasing our effectiveness.
Today as the world reflects on rare diseases, we are grateful to bring the spotlight to our fight against Duchenne. We are also grateful to each of you who support our fight and share our story. Because of you more people know about Duchenne. Because of you, we know we will one day be able to say that Duchenne is a disease of the past.