Paul Harmatz, MD

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges in investigating therapies for rare diseases.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed the dose response seen with RGX-121.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges with clinical trials in neurocognitive diseases including MPS2.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed biomarker findings from the phase 1/2 CAMPSIITE trial.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed updated data from the phase 1/2 CAMPSIITE trial presented at ASGCT 2023.