Myotonic Dystrophy 1 or DM1 is a genetic disorder affecting RNAs in a person DNA. Expansion Therapeutics dealing with this case has been trying to raise funds and have already raised $55.3 million in 2018. Led by 5 AM Ventures Managing Partner Scott Rocklage, Ph. D., several other companies such as Novartis Venture Fund, Kleiner Perkins, Sanofi Ventures, Alexandria Venture Investments and RA Capital Management have been involved in raising funds. DM1 does not have effective treatment methods necessitating the need for such efforts.
When some RNAs molecules from the DNA are transformed into proteins and accumulate, they reach toxic levels resulting into DM1. This affects the hormonal system, the heart, the respiratory system, muscles, and central nervous system rendering them inefficient. DM1 affects entire families with each following generation becoming worse.
Expansion therapists have given priority to expansion repeat diseases, which are inherited disorders that do not respond positively to updated medical interventions. Scott Rocklage when issuing a press release announced Matthew D. Disney as the lead scientist of the team. Dr. Disney said they are on the verge of producing small molecule treatments targeting the RNAs. This will be a significant breakthrough for patients who currently have no options. Scott Rocklage, Dr. Disney, and Expansion Therapist shave devoted their research until they come up with a treatment that will give hope for DM1 patients.
Scott Rocklage is a widely experienced medical health management professional with over 30 years experience in the field. His medical treatments endeavors have led to FDA approval of Cubicin, Teslascan and Omniscan medications in addition to other submissions of other medicines for clinical trials. Scott is an inventor with over 30 patents some of them co-invented. Additionally, he had written over 100 publications that have been peer-reviewed.
Scott went to the University of California, Berkeley for his B.S in Chemistry and Massachusetts Institute of Technology (MIT) for his Ph. D. in Chemistry. He had a chance to work with 2005 Nobel Peace Prize winner in Chemistry Dr. Richard Schorck when studying at MIT. Scott Rocklage is a leader having been chairman, president, CEO of several companies such as NycomedSalutar, Cubist Pharmaceuticals, and Catalytica. He is the chairman of boards of Kinestral, Rennovia and Cidara.
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