The Cures Within Reach Industry Patient Impact Research Award is being presented to Timothy P. Walbert, Chairman, President and Chief Executive Officer of Horizon Pharma plc, for his commitment to pursuing treatments for rare diseases, and his belief that repurposing is part of the solution. Tim has guided Horizon from an idea to a successful biopharmaceutical company in a short period of time. Moreover, he and his team at Horizon are laser focused on the rare disease community.
The Janet Davison Rowley Patient Impact Research Award is being conferred upon Drs. Berish Rubin and Sylvia Anderson, who jointly direct the Laboratory for Familial Dysautonomia (FD) Research at Fordham University. Drs. Rubin and Anderson and their team discovered the gene mutation for Familial Dysautonomia (FD), a rare disorder that impacts the development and function of the autonomic nervous system in affected individuals because of the loss of functional IKAP protein. They were then able to restore IKAP function through repurposing 1) two bioactive agents from soy; 2) derivatives of Vitamin A and E; and 3) EGCG, a bioactive agent found in green tea. Their repurposing research has been published in the numerous journals, including Molecular Nutrition & Food Research.
The Golan Christie Taglia Patient Impact Philanthropy Award is awarded to the FRAXA Research Foundation, a nonprofit committed to finding a cure for Fragile X syndrome. Fragile X is a rare disease that is the most common known cause of autism worldwide. Dr. Michael Tranfaglia, FRAXA's co-founder, will be accepting this award on his organization's behalf. FRAXA has invested significantly in repurposing research, and Dr. Tranfaglia's son, who has Fragile X, is treated with a repurposed therapy.
The Cures Within Reach Industry Patient Impact Clinical Award, a new award this year, will be given to Dr. Lieven Lagae of KUL University Hospitals, a pediatric neurologist for his research on Dravet Syndrome, a severe seizure disorder. Dr. Lagae is a pioneer in research repurposing the drug fenfluramine for Dravet. This drug was part of the Phen-Fen diet drug that was taken off the market, and fenfluramine, which was helping Dravet patients, was no longer available. Dr. Lagae has doggedly fought to bring Dravet patients access to this drug, and is leading a worldwide effort to secure regulatory approval for fenfluramine so that it will help stop these debilitating seizures.