UMD-led Study Could Offer Hope to Pompe Disease Patients
Pompe disease is a rare genetic disorder that disables heart and skeletal muscles and can lead to early death if untreated. The only available treatment for the disease is enzyme replacement therapy that must be injected regularly, sometimes every few days, for life. The treatment can cost hundreds of thousands of dollars a year. Researchers from the University of Maryland developed a method that could make enzyme replacement therapy more efficient, less expensive and less frequent. A study on their new method was published on August 19, 2021, in the journal Chemical Science.
“This work holds great promise for a better, less costly treatment of the life-threatening Pompe disease,” said UMD Chemistry and Biochemistry Professor Lai-Xi Wang, the corresponding author of the study.
