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.
Featured photo of Hani Kushlaf, MD, provided by UC Health.
Impact Lives Here
The University of Cincinnati is leading public urban universities into a new era of innovation and impact. Our faculty, staff and students are saving lives, changing outcomes and bending the future in our city's direction. Next Lives Here.
Stay up to date on all UC's COVID-19 stories, or take a UC virtual visit and begin picturing yourself at an institution that inspires incredible stories.
Related Stories
‘Designer drug’ shows early neuroprotective signal in acute ischemic stroke
October 28, 2025
Medscape highlighted new trial results led by the University of Cincinnati's Eva Mistry that found an experimental drug shows promise in protecting injured brain cells for patients with acute ischemic stroke.
Meet the young talent reshaping Cincinnati’s startup scene
October 28, 2025
Five University of Cincinnati students were honored for their impactful entrepreneurship on the Cincy Inno Under 25 list. We’ll explain what makes each student – and their startup – stand out.
Is menstrual fluid ‘the most overlooked opportunity’ in women’s health?
October 27, 2025
The Guardian recently reported that period blood has long been thought of as ‘stinky and useless’, but startups are exploring using the fluid to test for a wide range of health conditions — including endometriosis.