A new clinical trial at University of Florida Health show that gene therapy improved respiratory function in Pompe patients and increased the time they could spend breathing on their own without assisted ventilation.
UF Performs first diaphragm pacing surgery on child with Pompe disease
Addylen Reeder loves bubbles, singing and is fighting Pompe disease
Australian Brad Gibson receives an experimental drug that researchers hope will reverse or prevent the muscle debilitating effects of Pompe disease.
Michael Stanzione became the first person with Pompe disease in the world to receive a diaphragm pacing system, enabling him to breathe freely for the first time in four years. UF surgeons implanted the system, allowing Stanzione to resume a fulfilling life at home instead of in the hospital room.
The first commercially available treatment in the United States for patients with late-onset Pompe disease was administered Wednesday, June 16, at the University of Florida. Pompe disease is a rare form of muscular dystrophy and has been the focus of a research program at UF for more than 10…