Yahoo: Researchers find a new treatment for rare muscular disease

UC research presented at national meeting reveals a new possible treatment for Pompe disease

University of Cincinnati researchers have found a newer, more effective treatment for Pompe disease that could become the new standard of care for the rare condition.

Rare diseases are sometimes the most difficult to treat because of a lack of research and fewer participants to study. An example would be those who have Pompe disease, a genetic condition when a body can't make a protein that breaks down a complex sugar, called glycogen, for energy. Too much glycogen builds up and damages muscles and organs. The disease causes muscle weakness and trouble breathing and can affect the heart and muscles.

Hani Kushlaf, MD, an associate professor in both the Department of Neurology and Rehabilitation Medicine and the Department of Pathology and Laboratory Medicine at UC and principal investigator of a study led at UC, will present findings on this new treatment virtually at the American Academy of Neurology on April 20. Kushlaf is also a UC Health physician and a member of the UC Gardner Neuroscience Institute. 

Read the full Yahoo story.

Read the UC News story.

Featured photo of Hani Kushlaf, MD, provided by UC Health.

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