When parents are told that their child has a disease that is as awful as Duchenne Muscular Dystrophy, there are plenty of reasons to be despondent. After all, this is a disease that is “incurable,” and largely known as “untreatable.” It has only been through relatively recent advances in palliative care and a growing body of knowledge about substances such as steroids that kids are growing into their twenties with the disease. No major drug company has ever made an investment in Duchenne Muscular Dystrophy, because despite being the world’s top genetic lethal childhood disease, it is still classified as rare – one out of 3,500 boys. Biotechs are just now beginning to “kick the tires” out of interest in a potential profit in Duchenne Muscular Dystrophy treatment. The overwhelming majority of funds raised in the name of benevolence for Muscular Dystrophy go toward care, not toward research. And the governmental commitment to Duchenne Muscular Dystrophy, while improving, is woefully inadequate for the challenge.
Why, then, are we filled with so much hope? The reason is that Duchenne Muscular Dystrophy is a disease with a simple biology. Flaws on one gene one protein. We’ve had our gene identified for 17 years. Thanks to the work of Dr. Hoffman and many others throughout the world, we know a great deal about exactly how – and why – this disease progresses. You can’t say that about heart disease, cancer, diabetes, or any of the other diseases that receive billions of dollars in research funding. And the simple reality is that several teams of researchers have already identified ways to stop this disease in its tracks in the laboratory.
What separates us from the marketplace? Only two things: time, and money. They are everything.