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Follow on Google News | ![]() FRAXA Research Foundation to Be Awarded for its Dedication to fragile XFRAXA Will Receive Award for Its Dedication to Repurposing Treatments for a Known Cause of Autism
Fragile X syndrome is a rare disease that is a known cause of autism worldwide. FRAXA was founded in 1994 after three new parents' sons were born with fragile X syndrome. Since its founding, FRAXA's goal has been to accelerate the pace of fragile X research. FRAXA also knows that repurposing treatments are a key part of the research equation: of the $25 million FRAXA has funded in biomedical research since its inception, $3 million was earmarked for more cost-effective repurposing research. In fact, Dr. Tranfaglia's son's fragile X is treated with a repurposed therapy. "Cures Within Reach is pleased to recognize FRAXA," noted GHRA Co-Chair Nicole Boice, CEO and Founder of Global Genes, a leading global rare disease patient advocacy organization. "Given how prevalent fragile X is as a rare disease, research for this disorder has been drastically underfunded. The commitment FRAXA has had in dedicating funding to repurposing research creates promise for many patients. For this and many other reasons, we are honored to award this organization for their innovation and forward thinking related to drug re-development." The rare disease focus at this year's GHRA commemorates Cures Within Reach's first repurposing success, which found that the drug sirolimus created a life-saving treatment for a rare disease called autoimmune lymphoproliferative syndrome. The four Global Health Repurposing Awards that will be presented will honor leaders in business, science, medicine and philanthropy. The awardees' achievements have made a difference in patients' lives through repurposing research that finds new uses for existing drugs, devices and nutraceuticals. "I am honored to receive the 2017 Golan Christie Taglia Patient Impact Philanthropy Award on FRAXA's behalf," Dr. Tranfaglia, FRAXA co-founder, remarked. "This award will continue to raise awareness of the key role repurposing treatments can play in treating disorders like fragile X and other rare diseases." The Golan Christie Taglia Patient Impact Philanthropy Award, recognizes an individual, group or organization that has created positive patient impact by advocating for patients or contributing to the growth of repurposing research through financial, operational or professional philanthropic support. Previous awardees include: Dr. Nicolas Sireau, with Professor Lakshminarayan Ranganath, AKU Society; Lori Melichar representing the Robert Wood Johnson Foundation; George and Judy Goldman, Goldman Philanthropic Partnerships; To attend the 2017 GHRA and honor all awardees, visit http://www.cureswithinreach.org/ ABOUT CURES WITHIN REACH Cures Within Reach (http://www.cureswithinreach.org) works to catalyze repurposing research to quickly and affordably improve patient lives. We accomplish this by connecting funders with researchers to jumpstart repurposing research clinical trials, by providing collaboration tools so repurposing stakeholders can work together more easily, and by pioneering alternative finance engines and incentives for repurposing research. Cures Within Reach's repurposing research projects have generated over a dozen "new" treatments making patient impact through off-label use in clinical practice or through a commercialization track. Cures Within Reach currently has 24 repurposing research projects either funded or approved for funding. Visit us at www.cureswithinreach.org or follow us via Twitter @CuresWReach, LinkedIn.com/ ABOUT CUREACCELERATOR™ Cures Within Reach built CureAccelerator (http://www.cureaccelerator.org) ABOUT FRAXA RESEARCH FOUNDATION Founded in 1994, FRAXA is a nonprofit, tax-exempt organization based in Newburyport, Mass. Committed to finding a cure for fragile X, FRAXA has funded more than $25 million in biomedical research, yielding discoveries that are changing the lives of families coping with fragile X. FRAXA is one of the most efficient and effective charities in the world, with management and general expenses under 4 percent and research expenditures at 86 percent. Three Nobel Laureates sit on its volunteer Scientific Advisory Board. End
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