Family holds out hope for a cure for a rare genetic disorder
Research at the Children’s Hospital of Wisconsin Research Institute and MCW is providing hope for families with children with rare muscular disease. Michael Lawlor, MD, PhD, assistant professor of pathology and director of the Pediatric Pathology Neuromuscular Laboratory, discussed gene therapy that has been effective in treating muscular disease in dogs with the same condition that affects humans. Dr. Lawlor is working with Jennifer Strande, MD, PhD, assistant professor of cardiovascular medicine; and collaborators at other institutions to get new therapies approved for children with MTM and other neuromuscular diseases.
WTMJ-TV, April 9, 2014