DM1 Muscle Issues Cured in Mouse Model

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.

Continue reading