He’s walked the red carpet and socialized with movie stars, but he’s also saved lives.
Dr. Barry Byrne is a pediatric cardiologist and director of the Powell Gene Therapy Center at the UF College of Medicine.
He is spending time in the spotlight while his research on Pompe disease, a rare genetic disorder resulting in infant mortality, is showcased in the new movie “Extraordinary Measures.”
Byrne said the movie, which describes a father’s efforts to find a cure for his children with Pompe disease, has a simple message.
Byrne said he met with producers of the film as well as its stars, Harrison Ford and Brendan Fraser.
As shown in the movie, the disorder causes paralysis and can force patients to breathe with a respirator. The patient’s heart eventually shuts down.
Byrne participated in the clinical trial that led to the approval of Myozyme, the first form of enzyme replacement therapy for Pompe disease and the treatment featured in the movie.
Byrne is also working on clinical trials for gene therapy of Pompe disease.
Byrne said gene therapy is the treatment that he and his team have the best hope for, and they are gearing up for human trials of the treatment.
