Myotonic Dystrophy may be the first of the muscular Dystrophy to have a definitive cure established. In the last few years researchers have isolated the issue they believe causes myotonic dystrophy. In the last couple of years therapies that have targeted this problem have reversed the disease in cell and animal models. This has set up a race now for researchers to refine the therapeutic compounds and begin with human trials to see if the animal to human therapeutic transfer works as it is believed to. Thus, this would lead to the first potential cure for a type of muscular dystrophy.
The Myotonic Dystrophy Foundation (MDF) in Menlo Park, CA has taken a lead in assisting researchers with moving forward with this disease elimination process with funding and assistance in establishing a patient registry. The MDF was the early funder of a new Research Collaborative effort lead by Charles Thorton out of the University of Rochester. The foundation has also been instrumental in educating patients and physicians in this complex and slowly progressing disease that results in premature death. A patient conference is planned for early November of 2013 in Houston, TX
Exciting news. This summer researchers at ISIS Pharmaceuticals were able to treat and Cure DM1 in a mouse model (A mouse model is a mouse that has been genetically altered to have the symptoms of DM1) This is really exciting news. The treatment seemed to work well and was by injection. The injection seemed to work long term as well. Next they will most likely use a Monkey and then if that is successful with Humans! This is 5-8 years off and will need millions of dollars of research.
The payoff for these companies will be huge though. With over 42,000 people in North America alone the market size is roughly $1 Billion for the USA. If the injections prove successful a once a year or 2x per year injection would lead to a low-cost of goods sold and high profitability. Worldwide at $20,000 per dose this is about a $17Billion dollar market.