The following is an excerpt.
Children with a rare form of muscular dystrophy called Pompe disease often spend their days tethered to mechanical ventilators in order to breathe. But results from a new clinical trial at University of Florida Health show that gene therapy improved respiratory function in these patients and increased the time they could spend breathing on their own without assisted ventilation.
Read the full story here: Gene Therapy Helps Muscular Dystrophy Patients Breathe Easier