PHILADELPHIA – A Penn professor’s research efforts to fight debilitating degenerative muscle diseases have earned him international recognition by the Deputy Ruler of Dubai and Minister of Finance and Industry of the United Arab Emirates.

H. Lee Sweeney, PhD

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In November 2008 Sheikh Hamdan Bin Rashid Al Maktoum named H. Lee Sweeney, PhD, William Maul Measey Professor at the University of Pennsylvania School of Medicine, as one of three recipients of the Hamdan Award for Medical Research Excellence. The award honors individuals whose medical research has helped to improve the lives of thousands of people worldwide.

Dr. Sweeney, the sole US recipient of this year’s award, was recognized for his research on muscular dystrophy, with emphasis on the impact in the area of pharmacogenomics, the science of how genetic variations influence individual differences in responses to drugs.

“This award recognizes a number of contributions I have made in the area of combating muscle weakness and muscular dystrophy, including my recent role in the development of a drug which may be able to treat a subset of patients with muscular dystrophy and other genetic disorders,” Sweeney says. “It was a great honor and surprise to have my work recognized while the clinical development of [such drugs] is ongoing.”

For much of his career, Dr. Sweeney has studied the mechanisms that help control muscle function with the hope of gaining a better understanding of ways to thwart muscle deterioration caused by age and degenerative diseases and promote muscle growth.

Dr. Sweeney’s research includes the study of animal models of Duchenne’s muscular dystrophy (DMD), a rapid muscle degenerative disease that can lead to paralysis of all muscles in the body, including the heart and diaphragm, and death. His research team at Penn uses gene therapy, a method where new genes are inserted into cells, to help treat genetic diseases as a means to validate possible therapeutic targets.

Beginning in 1999, Dr. Sweeney’s lab published the first paper showing that a family of antibiotics known as aminoglycosides could be used to suppress disease-causing genes in DMD mice. In 2007, Dr. Sweeney then led a team of researchers who showed a new drug called PTC124 could override a genetic mutation causing muscle degeneration in DMD mice without causing side effects. PTC124, developed by PTC Therapeutics in collaboration with Dr. Sweeney’s lab, is currently in the pivotal clinical trial phase in DMD patients.

As part of the Hamdan Award for Medical Research Excellence Dr. Sweeney was invited to present a lecture at the 5th Dubai International Conference for Medical Sciences last December, where he discussed his most recent studies.

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