Scott Rocklage Is Working Towards Treating DM1 With Small Molecules

Scott Rocklage achieved his education at the University of California, Berkeley. He graduated with his Bachelor of Science in Chemistry. He then attended the Massachusetts Institute of Technology where he earned his Ph.D. in Chemistry. He conducted his research in the laboratory of Richard R. Schrock while attending MIT. In 1985 Richard R. Schrock was awarded with the Nobel Peace Prize for his work in chemistry.

Dr. Scott Rocklage has spent in excess of thirty years in the sector of healthcare management. His hard work resulted in approval from the FDA for three separate medications. He has also submitted additional medications for clinical trials. He possesses over thirty patents and has had well over 100 publications reviewed by his peers. Dr. Scott Rocklage is a leader in his field.

Expansion Therapeutics has been working towards battling genetic disorders. Myotonic dystrophy 1 type is the prevalent cause of adult muscular dystrophy. The company is spending $55.3 million to battle this disease. 5AM Ventures along with Kleiner Perkins and the Novartis Venture Fund all participated in the financing.

Molecules converted into proteins after a DNA transfer are called RNA’s. When the production of these molecules becomes toxic within the cells DM1 occurs. This causes defects in the heart, gastrointestinal system, respiratory system, muscles, hormonal system, and the central nervous system. DMI often affects complete generations and becomes worse with the passage of time and the new generations. This disease does not have an effective treatment available at this time.

The research performed by 5AM’s Managing Partner Scott Rocklage and Matthew Disney at the laboratory is used by Expansion Therapeutics. It has become apparent small molecule medications can be used to address diseases related to RNA. The treatments required by the individuals with the most crucial medical needs are getting much closer. The team plans to continue until they are successful. Expansion Therapeutics and Dr. Scott Rocklage are working together so the small molecule medical field can be advanced to target RNA. Their work is important in the creation of medications for patients with no options. These medications can potentially treat illnesses triggered by RNA.


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