Scott Rocklage: Pioneer in Muscular Dystrophy Cure

A company called Expansion Therapeutics created a financing system to battle varying genetic dysfunctions. One type of this disorder is the Myotonic Dystrophy Type I, which is considered one of the main causes of Muscular Dystrophy.

Expansion Therapeutics funded the research to a worth of $55.3 million to help find a cure for this disease, according to Dr. Scott Rocklage, in a press release. Some signs of the disease are noted to exist when RNA incurs a toxic amount. Learn more about Scott Rocklage: and

The companies that additionally funded the research are Novartis Venture Fund, Sanofi Ventures, Kleiner Perkins, 5AM Ventures, Alexandria Venture Investments and RA Capital Management.

The Ribonucleic Acid or RNA are molecules that are transported from the DNA and eventually turned into proteins. Muscular Dystrophy happens when the RNA production is noted to reach high levels and product toxicity in the cells.

This is particularly known as the DM1 condition. The result is a defective muscle state, heart problems, gastrointestinal issues, hormonal imbalance and even central nervous system affectations.

Expansion Therapeutics has devised a way to target the RNA for conditions which etiologies are caused by this molecule. One of the company’s main focus is the treatment of diseases that have expansion repetition disorders.

These are the type of diseases that can run through generations of families, do not respond to traditional treatments, and can attack an individual at a certain gender or age. This type of diseases may be caused by mutated genes, which are repeated portions of the original DNA. The DM1 condition of muscular dystrophy is considered a variation of expansion repetition disorders.

The research performed by Matthew D. Disney is the basis of this treatment. Managing partner Scott Rocklage of 5 AM Ventures also released a press statement about the treatment that the company is trying to create.

About Scott Rocklage

Scott Rocklage is 5AM Venture’s leading managing partner. He earned a Bachelor of Science in Chemistry at University of California, Berkeley. Eventually, he earned his Ph.D. in Chemistry at Massachusetts Institute of Technology. During his time in MIT, he started a research along with Richard R. Schrock in his laboratory. Dr. Schrock won a Nobel Peace Prize in 2005.

Dr. Scott Rocklage became part of 5AM Ventures as a Venture Partner in 2003. He rose to become a Managing partner a year after his work with 5AM Ventures.

His 30 years of experience in healthcare has allowed him to obtain FDA approval of three medications: Omniscan, Cubicin, and Teslascan. He is an innovative researcher and has patented over 30 products that he has personally created or made with a team. He also has more than 100 research journals published and reviewed by peers.

Read more: Scott Rocklage | Bloomberg and Scott Rocklage | Crunchbase