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Kansas City, Kansas and Paris, France. What do they have in common?

Both cities are the only locations in the world conducting similar study on rare muscular disease.

Kansas City has long been called the “Paris of the Plains” due to boulevards, water fountains and strong cultural engagement. Now Kansas City is even more connected to Paris as one of two locations in the world leading a stem cell study on a rare muscular disease.

Mazen Dimachkie, M.D., professor of neurology and director of the neuromuscular division at the University of Kansas Medical Center, is the principal investigator of the study in Kansas City. The research focuses on adipose derived stem cells in a rare muscle disease, called inclusion body myositis (IBM).

IBM is a rare condition that causes muscle weakness and damage. Symptoms of IBM vary, but usually include progressive weakness in muscles of the hand, forearm, thigh and lower leg. Diagnosing IBM can be challenging because the symptoms are not unique to this condition.

Also, IBM is often undiagnosed or misdiagnosed, so surveys may not discover all the actual cases. An estimated 25,000 people in the U.S. have IBM.

The primary endpoint of the trial is safety of study subjects by reviewing side effects after collection of fat tissue and the injection of the cell product in IBM patients. “We hope to see signals of efficacy that the injections either slowed, halted or improved muscle function in the injections to the forearm and thigh muscle,” said Dimachkie.

KU School of Medicine

University of Kansas Medical Center
Department of Neurology
Mailstop 2012
3901 Rainbow Blvd.
Kansas City, KS 66160
Phone: 913-588-6970
Fax: 913-588-6965