Paul Harmatz, MD, on Challenges With Assessing Neurocognitive Outcomes
The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges with clinical trials in neurocognitive diseases including MPS2.
Paul Harmatz, MD, on Assessing D2S6 in Trials for MPS Type 2
The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed biomarker findings from the phase 1/2 CAMPSIITE trial.
Paul Harmatz, MD, on Reducing GAGs in MPS Type 2 With RGX-121 Gene Therapy
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.
