On February 7, 2013 our leading researcher in the fight against myotonic muscular dystrophy, Charles Thornton, was installed as the inaugural Saunders Family Distinguished Professor in Neuromuscular Research at the University of Rochester. You can read the full article here (about half way down the page).
As described by Mark Taubman, University of Rochester dean of the School of Medicine and Dentistry, “Charles epitomizes the translational scientist. His current work is taking advantage of exciting new developments in RNA biology to provide novel approaches to crippling diseases. And as an active clinician, he is ideally suited to be able to take results from the lab to the patient, which is exactly the work being done in the Saunders Research Building.” As highlighted in previous posts on this blog, we’ve seen this to be true firsthand and are thrilled to see Charles receive this recognition.
WESTON, Mass. & CARLSBAD, Calif.–(BUSINESS WIRE)–Jun. 29, 2012– Biogen Idec (NASDAQ: BIIB) and Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today announced that they have entered into an exclusive, worldwide option and collaboration agreement under which the companies will develop and commercialize a novel antisense drug for the treatment of myotonic dystrophy type 1 (DM1), which is also known as Steinert disease. (from press release)
Given the Thanksgiving holiday we figured it was high time to give thanks to everyone who has contributed to Run America – both in dollars and sweat! Our apologies for the time that has passed since our last update, but it was worth the wait as we have some incredible news that became public this past summer: “Entrepreneur and philanthropist Philip Saunders made history with his donation of $10 million to the neuromuscular disease program of the UR Medical Center — one of the largest gifts in the institution’s history.”
Neurologist and Director of the Neuromuscular Disease Center Richard Moxley, who has worked closely with Barry for many years, was the first to get the great news and commented that “we have targeted myotonic dystrophy because it has the greatest promise of being a disease that we can reverse.” The story of how this came about is worth a read (link at bottom) and the stories that may come out of it will surely bring many more “thanks givings” in the years to come.
No news yet on Run America V but we’ll keep everyone posted! A happy, healthy holiday season to all.
Recently, Dr. Charles Thornton from the University of Rochester Medical Center, who leading the research team our fund-raising efforts support, shared some exciting news about the team’s efforts to find a cure for myotonic muscular dystrophy. Paraphrasing a bit he explained: Earlier this year we applied for a large grant of around $5.6 million over 4 years. The scientists who reviewed the project gave it high marks and it had to go through administrative review at NIH (National Institutes of Health). In late September we learned the grant will be funded. The goal of the project is to optimize a drug that can be taken forward into clinical trials, and to do all of the safety testing that is needed to obtain an “IND” – permission by the FDA to give a new drug to people. The money goes mostly to Isis Pharmaceuticals to cover the costs of purchasing raw materials and, most expensively, to subcontract with the laboratories that do the animal safety testing, which is very extensive. The optimization parts are done jointly at Isis and University of Rochester. Having this co-investment from the NIH, (about 40-50% of their development costs over the next 4 years) helps to make sure that the work will go forward.
Shortly after learning the news, Dr. Thornton was the first researcher to speak at a joint NIH/FDA conference called “Antisense Oligonucleotides in Neuromuscular Disease.” It was the first meeting of its kind. He was glad to acknowledge that their work was supported by NIH and went on to acknowledge “that a small group called Run America Foundation helped get the whole thing launched.”
We’re very hopeful this work will lead to a breakthrough medication that can slow down, stop or even reverse the debilitating effects of myotonic muscular dystrophy. Thanks to everyone who contributes financial and moral support to our cause. Happy Holidays!
With the days getting shorter and the mercury falling along with the leaves, it seems like a lifetime ago that we completed our trip around Lake Michigan!
The gang on the final morning of Run America IV
Here in the Thanksgiving season it seems particularly appropriate to thank all the Run America donors who together have contributed just shy of $15,000 (literally we just need another $46 as of this writing!), all of which goes toward continued research to find a cure for myotonic muscular dystrophy. This $15,000 brings our total over the past eight years and four events to $350,000 – an incredible sum that is generating real results. We’ll be posting some exciting news on how that money is being put to use soon.
Thanks again for your time, energy and generosity!
The whole team of runners and bikers is getting excited about our upcoming trip, Run America IV. Bikes are being shipped and running shoes replaced as we prepare for our 947 mile journey around Lake Michigan.
A big thanks to everyone who has donated thus far–we hope to reach $10,000 by the time we start–and we appreciate everyone spreading the word so we can continue to raise funds for research to fight myotonic muscular dystrophy. We’d also like to thank Catrikes and Clif Bar for stepping up as product sponsors.
We are hoping to raise awareness during our trip as well so we’d welcome any and all press opportunities. Please contact us if you’ve got any ideas. See you us if you’ve got any ideas. See you on the road!
Registration is now open for Myotonic Dystrophy Foundation (MDF) family conference, Empower, 2010 Myotonic Dystrophy Family Conference, August 13-15, 2010. This conference will bring important research updates, information about diagnosis, treatment, and daily living issues to assist individuals living with myotonic dystrophy, families, health care providers and the community. Learn more here!
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About Run America
We are a group of friends completing endurance running and biking events to raise funds for research dedicated to finding a cure for myotonic muscular dystrophy, the most common form of muscular dystrophy affecting infants and adults. Learn More.
Run America is an ongoing series of elite endurance events intended to raise money for myotonic dystrophy research. Myotonic dystrophy is the most common adult form of muscular dystrophy. The goal of Run America is to provide funds to seed promising research efforts for therapies and an eventual cure for this genetic disease. We are working in partnership with the Myotonic Dystrophy Foundation, a non-profit organization whose mission is to lead and mobilize resources toward effective management, treatment, and an ultimate cure for myotonic dystrophy through education, advocacy and research.