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

United statesHani kushlafEmily hendersonDepartment of neurologyDrug administrationAmerican academy of neurology onUniversity of cincinnatiLaboratory medicineDepartment of pathologyUc healthUc gardner neuroscience instituteRehabilitation medicineAmerican academyஒன்றுபட்டது மாநிலங்களில்எமிலி ஹென்டர்சன்துறை ஆஃப் நரம்பியல்