Page 3 - Neuromuscular | UR Medicine

Closing in on a Cure

For decades, Drs. Charles Thornton, Richard Moxley, and teams of UR Medicine researchers have studied myotonic
dystrophy, hoping to someday spot its Achilles’ heel. Their work has come close to doing that. Thornton and Moxley

have been recognized by the Myotonic Dystrophy Foundation with an Outstanding Research Achievement Award
in recognition of the enduring and transformative collaboration that they have carried out in myotonic dystrophy
research and clinical care, and the outstanding progress they have made possible in the search for new treatments and
a cure for the disease.

This recognition follows on the heels of a $7 million grant from the National Institute of Neurological Disorders and
Stroke (NINDS) to renew funding for the University’s Senator Paul D. Wellstone Muscular Dystrophy Cooperative
Research Center, a designation that dates back to 2003. The team is also preparing—in collaboration with Isis
Pharmaceuticals—to begin testing the first targeted treatment for the disease.

The collaborative nature of UR Medicine has put our researchers at an advantage to understand the complexities of
myotonic dystrophy. Thornton, Moxley, and their colleagues have used a different approach to translational research—
using team science to unite several lines of research, all aimed at myotonic dystrophy, in a single program. This

includes basic genetics, drug development, preclinical drug testing, and clinical trials. This concerted environment,
where research and clinical practice each strengthens the other, has put us in a uniquely powerful position to tackle
this complicated disease. Combined with the tireless commitment and participation of patients and their families, we
are well on our way to closing in on a cure.

“Phil Saunders’ gift ensures that our people have the freedom to pursue multiple promising projects

simultaneously as we seek better treatments for conditions like muscular dystrophy”
Richard T. Moxley, III, M.D., the Helen Aresty Fine and Irving Fine Professor in Neurology (right).

In 2011, Philip Saunders (left), a Rochester businessman and a major
supporter of the UR Medicine neuromuscular disease program, donated
$10 million to the program as well as other UR Medicine research
initiatives. The Saunders Research Building was named in his honor.