Cure Duchenne, a national, non-profit dedicated to funding research and raising awareness for Duchenne, the most common and devastating muscle disease in children, will host its 2nd annual Duchenne Summit at the Newport Beach Marriott Hotel, 900 Newport Center Drive, Friday, January 28 from 8 a.m. to 5 p.m. and Saturday, January 29 from 8 a.m. to noon.
The Duchenne Summit brings together the top Duchenne scientists, biotech executives, regulatory experts and physicians from around the country to develop research strategy for the coming year. Those scientists serving as scientific advisors at this year's Summit and their affiliations are:
- Barry J. Byrne, M.D., Ph.D.
Professor & Associate Chair of Pediatrics, Molecular Genetics & Microbiology Director, Powell Gene Therapy Center, University of Florida, School of Medicine
- Kevin P. Campbell, Ph.D.
Investigator, Howard Hughes Medical Institute; Director, Senator Paul D. Wellstone MDRC, University of Iowa, Carver College of Medicine
- Jeffrey Chamberlain, Ph.D.
Principal Investigator, Chamberlain Lab Department of Neurology, University of Washington School of Medicine
- Eric Hoffman, Ph.D.
Director, Research Center for Genetic Medicine, George Washington School of Medicine and Health Sciences, Children's National Medical Center, Washington, DC
- Edward Michael Kay, M.D.
Group Vice President, Clinical Research Genzyme
- Douglas Macdonald, Ph.D.
Director, Drug Discovery, CHDI Management, Inc.
- M. Carrie Miceli, Ph.D.
Professor, Departments of Human Genetics, David Geffen School of Medicine at UCLA
- Stanley F. Nelson, M.D., Ph.D.
Assistant Professor, Director of Pediatric Neuromuscular Clinic
- Pier Lorenzo Puri, M.D., Ph.D.
Assistant Professor, Burnham Institute for Medical Research, San Diego, Calif.
- Brian Tseng, M.D., Ph.D.
Assistant Professor, Director of Pediatric Neuromuscular Clinic, Mass General Hospital, Harvard Medical School, Boston, Mass.
Based, in a large part, upon the scientist's recommendations, CureDuchenne then contributes to the national and international research showing the most promise. Previous year's contributions from Cure Duchenne have resulted in six research projects now in human clinical trials.
On day one of the Summit (Friday, January 28), CureDuchenne's scientific advisors will hold a confidential session to review and analyze all possible research projects targeting Duchenne. Debra Miller, founder and president of Cure Duchenne, says, "Our scientific advisors are the leading experts on Duchenne and they play a key role in our success. We believe the model we have created in the Duchenne community provides the most comprehensive review across all potential treatment areas because of the expertise of our advisors in gene therapy, exon skipping, pharmacologic therapies, biotech and pharmaceutical and regulatory issues."
On day two (Saturday, January 29), from 8 a.m. to noon, Cure Duchenne will host the "Breakfast with the Experts." The session includes a briefing from the advisors and a question and answer period. The session is open to the general public. Duchenne families, advocates and those who are interested in attending may register at http://www.cureduchenne.org/site/Calendar?id=100082&view=Detail. The cost is $35 per person and includes breakfast. The session is also being offered to those who cannot attend via a free webcast at ustream.tv/channel/cureduchenne.
Cure Duchenne is a 501(c)3 national, nonprofit located in Newport Beach, Calif. The organization was founded in 2003 by Paul and Debra Miller after their son and only child was diagnosed with Duchenne Muscular Dystrophy. CureDuchenne has established itself as a leader in funding successful research projects. It was the lead organization funding Prosensa, a Dutch biotech company that received a commitment of up to $650 million from Glaxo Smith Kline – making it the first time a major pharmaceutical company has invested in Duchenne. More information on Cure Duchenne can be found at cureduchenne.org.
Contacts:
CureDuchenne
Peggy Larson
949-872-2552
peggy@cureduchenne.org