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
In 2003 Dr. Scott Rocklage became a founding partner at 5AM Ventures. Today, he is a Managing Partner. 5AM Ventures provides start-up capital for businesses that develop research instruments and biopharmaceuticals, as well as financing research medical technology and life sciences research. Dr. Scott Rocklage was the CEO of Cubist Pharmaceuticals. He was both president and CEO of Nycomed Salutar.
Myotonic Dystrophy Type 1 (DM1) is a genetic condition that can lead to adults developing muscular dystrophy. DM1 affects breathing, the brain, spinal cord, hormones, endocrine system, the digestive system, and heart.
If a parent passes on the DM1 mutation the gene their child carries will be stronger than the parent’s DM1 gene. In the child’s offspring, the gene will be stronger still. As of this writing, there is no treatment for DM1.
A situation that 5AM Ventures and five other venture capital firms including RA Capital Management and Novartis Venture Fund have devoted $55.3 million to correct. The mutation in the Myotonic Dystrophy Type 1 gene is the result of malfunctioning RNA. RNA transmits messages from the DNA to cells to regulate the synthesis of proteins.
For over a decade a research team led by Dr. Matthew Disney has been researching hereditary illnesses for which no effective treatment is available. These diseases which include DM1 are called Expansion Repeat Disorders. The perpetuation of a defective portion of DNA leads to overproduction of RNAs. Toxicity results when too much RNA has built-up.
Dr. Disney and his team’s strategy involves the use of Small Molecule Medications. Small Molecule Medications control biological processes. These meds are called Expansion Therapeutics. The treatment will specifically target the RNA causing the disease.
Dr. Disney expressed optimism that help for those suffering from Expansion Repeat Disorders is not far off.
Dr. Scott Rocklage has worked in the Pharmaceutical Industry for 30 plus years. He has a BS in Chemistry from UC Berkeley. His PhD. in Chemistry is from MIT. He holds or co-holds more than 30 patents. Dr. Rocklage is responsible for the FDA approving Cubicin a treatment for skin infections and the MRI contrasting agent Omniscan.
Connct with Dr. Scott Rocklage on LinkedIn.
The education secretary Betsy DeVos was selected by President Trump for her work pushing for school vouchers and charter schools. She has additionally long been a republican chairwoman and a big donor in the state of Michigan. Who is Betsy DeVos though? There is much more to the education secretary then may be expected.
Education secretary Betsy DeVos is generally seen as a polite woman who comes from a wealthy billionaire family. The DeVos family are involved in political donations to the Republican party. DeVos herself has extended her interest in the affairs of her state beyond the political with her involvement in the charter and private school education system in the state of Michigan. Her interest in school vouchers and pushing for charter schools made her a shoe in for secretary of education in President Trump’s campaign. While Ttump himself is a larger than life figure in current politics, it may be expected that a polite, well to do woman like DeVos would go along with everything the president decides. Betsy DeVos has never been one to stick to polite niceties when she cares about something in the political world.
Although DeVos is mainly loyal to the president so as to help him further his agenda concerning the education system in the United States, she has stuck true to her own convictions as well. DeVos vocalized opposition to the Trump administration removing trandgender protections for students. She told Attorney General Jeff Sessions first of all about her issue with the decision on the part of the Trump administration to remove protections for transgender youth in school. Ultimately, DeVos went along with the Trump administration’s decision. Her decision to vocalize concern about the safety of students in the school system did result in the Trump administration rephrasing the letter of removal of the transgender policy removal and DeVos herself stating that the administration continues to be concerned about the safety of students. DeVos views the transgender policy and other education policy passed during the Obama years to be a clear sign of the overreach the Obama administration had over the school system. She stands with the political point of view that new policy must be put into place to protect students without having an overreaching enforcing arm over school policy.
While DeVos may be polite on the outside and the final result of her political efforts is often in a polite manner, she does know how to push firmly and strongly for her political point of view to be heard. Betsy DeVos and her family will continue to be a part of the political efforts of the conservative Republican Party in the United States. No doubt the American people will only continue to hear about Betsy DeVos and her influential family.
To learn more, visit http://www.betsydevos.com/.