Mytonis Dystrophy type 1 a genetic disorder that deteriorates muscles and organs until they no longer function. The disease affects about 1 in 8,000 people in the world and typically appears between the age of 20 and 30. The disorder is commonly referred to as Steinert disease. Learn more: http://medicaldailytimes.com/medical-research/scott-rocklage-leading-way-cure/3159/
Scott Rocklage and his organization, Expansion Therapeutics, have been attempting to find cures and therapies for patients with DS1. In 2018 the company announced that it had raised an additional 55 million dollars to help fund the way to helping patients.
The rare disease occurs when fragments of DNA repeat themselves in an individual. These cloned sequence of DNA are the cause of RNA increase, which in turn, due to its toxic properties in high levels begin the muscle deterioration. Rocklage and Company are working to create medicines that attack the RNA and put a stop on the damage that mytonis dystrophy has caused. There is no known cure at the moment, but everyone at Expansion Therapeutics are excited to adventure into the unknown and possibly provide relief for thousands of hurting patients.
About Scott Rocklage
Scott Rocklage was a student at the University of California, MIT, and Berkeley and is now a Managing Partner at 5AM Ventures. Rocklage joined the company in 2003 and in just two years he was awarded the Nobel Peace Prize in Chemistry.
Before joining 5AM Ventures Rocklage spent two decades with other companies producing new FDA approved medicines. He has worked with companies such as Cubist Pharmaceuticals, Nycomed Salutar, Relppsa, and Achaogen. Scott Rocklage currently resides in Boston, Massachusetts