GAINESVILLE, Fla. — As scientists work to find new treatments for Pompe disease — the devastating genetic "villain" that drives the efforts of the main characters in the new film "Extraordinary Measures" — University of Florida researchers are hopeful that gene therapy will help patients in the late stages of the disease breathe on their own.
Clinical trials of a gene therapy for Pompe-related breathing problems in six infants are expected to begin at UF this summer.