The Story of Expansion Therapeutics: Scott Rocklage Paves the Path for Adult Muscular Dystrophy Cure

Scott Rocklage, Ph.D., founding investor of Expansion Therapeutics, is part of a drug development company among others that raised $55.3 million to treat genetic abnormalities. This benefits patients with myotonic dystrophy type I (DM1), the top cause of adult muscular dystrophy.

This genetic disorder disturbs entire families, and can grow worse and worse with each following generation.

Inside the cells, the organic molecules transformed from DNA which turn into protein, or RNA, are surmised to cause symptoms of DM1 when they reach levels the body deems as toxic.

Eventually this leads to whole-body disorders. Including, but not limited to: irregularities in the heart, muscles, gastrointestinal system, hormonal system, central nervous system and respiratory system.

Expansion Therapeutics is spearheading their involvement on “expansion repeat disorders” (genetic forms of illnesses without a cure). These disorders appear as short DNA portions duplicate. Learn more about Scott Rocklage: and

This includes DM1 along with 30 other small molecule medications they are focusing on. As more and more DNA repeats, it boosts RNA reproduction which accumulate and reach toxic amounts.

Research is performed at Matthew D. Disney’s lab. Scott M. Rocklage, Ph.D.,Managing Partner of 5AM Ventures, stated that Dr. Disney is the main scientific researcher on the team. Everyone involved is delighted their work over the past 12 years has led them their current direction. They pledge never to stop until those with the direst needs are fulfilled.

Expansion Therapeutics have a goal to advance the field of small molecule drugs, and are eager to continue collaborating on medications for patients with sub-optimal treatments.

Beth Seidenberg, M.D., Partner of Kleiner Perkins, is thrilled to partner with Expansion and others to identify patient specific therapies, using science that can potentially treat diseases like DM1.

About Scott Rocklage

Scott became a Managing Partner for 5AM Venture in 2004, and with more than 20 years of strategic experience in healthcare and leadership roles, Dr. Rocklage has achieved approval for Omniscan™, Teslascan® and Cubicin® by the FDA.

Dr. Rocklage earned his B.S. in Chemistry from U.C. Berkeley, and his Ph.D. from MIT in Chemistry and currently serves as Board of Chairman for more than 10 different companies

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