World Leader in the Search for New Treatments for a Rare Disease

Elif A. Oral, M.D., M.S.Lipodystrophy is a rare, currently incurable syndrome tied to a number of diseases, and can lead to diabetes and very high triglyceride levels. Life-threatening complications are caused by a decrease in the hormone Leptin, and result in symptoms like uncontrolled body fat loss or accumulation, and a stressed liver.

Elif A. Oral, M.D., M.S. currently has the largest lipodystrophy patient population in the United States, and spearheaded the development of the only approved therapy for the condition. Dr. Oral is collaborating with pharmaceutical multiple companies– Aegerion, Regeneron, Akcea Therapeutics, and Gemphire Therapeutics–on new therapies, and is the principal investigator on world-wide clinical trials, with U-M serving as the main trial site. She was also a co-organizer of “Lipodystrophy Symposium 2018: A Call for Global Action,” an international conference lead by Michigan Medicine that included clinicians, researchers, patients, and companies.

“Ultimately, we’re striving for better patient care and cures. Companies like Aegerion and Regeneron help us achieve those goals, getting our treatments to market more quickly. And our expertise and access to patients help the companies bring their innovations to the correct patients far more quickly,” notes Dr. Oral. “The University of Michigan’s reputation, resources, and network enable us to connect with potential collaborating companies in ways that would be impossible anywhere else.”

Groundbreaking work in a rare disease like lipodystrophy is just one example of how U-M Medical School researchers are accelerating new treatments and technologies into the marketplace.  In 2018, we had over one thousand funded projects from industry partners like pharmaceutical companies and device manufacturers. We are in the fifth year of our collaboration with the Michigan Economic Development Corporation, this year providing over $2 million in state funding for early-stage projects–here at the U-M and across the state–that are on their first steps towards commercialization. And, our commercialization education program teaches the ins and outs of the business world, so our academic researchers of today can turn into the serial innovators of tomorrow.

Patient Valerie Walton is shown above with Elif A. Oral, M.D., M.S., Associate Professor in the Division of Metabolism, Endocrinology & Diabetes and Medical Director of the Bariatric Surgery Program, Director of the MEND Obesity and Metabolic Disorder Program, and Director of the Post-Bariatric Surgery Care Program.

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