Fuller and Byrne Awarded NIH R01 for Pompe Disease

Dave Fuller, Professor of Physical Therapy and BREATHE Associate Director, and Barry Byrne, Associate Chair of Pediatrics, Director of the Powell Gene Therapy Center, and BREATHE member, received a 5-year NIH R01 award to continue their work on gene therapy for Pompe disease, an inherited disorder that can eventually lead to progressive muscle weakness, including the muscles that control breathing. This is a competitive renewal of an NIH grant that Drs. Fuller and Byrne have held for the last 10 years. This collaboration has produced 25 published manuscripts and contributed to an initial clinical trial of diaphragm-directed gene therapy in children with Pompe disease (NCT02354651). The new grant award focuses on systemic AAV therapy for cardiorespiratory dysfunction in Pompe disease. Congratulations to Drs. Fuller and Byrne!