► 13:25 An Overview of Research Program for Pompe Disease Pompe Disease Content Hub Priya Kishnani, MD, of @DukeMedSchool, discusses #translational #research on #PompeDisease.
► 1:37:47 Small Molecule Inhibitor Reduces Glycogen in Preclinical Mouse Models of Pompe Disease Pompe Disease Content Hub Julie Ullman, PhD, of @MazeInBiotech, discusses #preclinical #results on #MZE001 for #PompeDisease.
► 02:10 Multidisciplinary Approach to Managing Pompe Disease Pompe Disease Content Hub Priya Kishnani, MD, of @DukeMedSchool, discusses #multidisciplinary care for #PompeDisease.
► 54:32 Newborn Screening for Pompe Disease Pompe Disease Content Hub Damara Ortiz, MD, of @ChildrensPgh, discusses #screening for #PompeDisease.
► 12:20 Phase 3 Trial Data for Next-Generation Enzyme Therapy in Pompe Disease Pompe Disease Content Hub Priya Kishnani, MD, of @DukeOTC, discusses #treatment for #PompeDisease.
► 05:49 Addressing Loneliness and Isolation in Pompe Disease Pompe Disease Content Hub Dr Sue Hawkins discusses how to combat #loneliness and #isolation in #PompeDisease.
► 2:41 Use of Electronic Health Records to Assess Risk for Lysosomal Storage Disease Pompe Disease Content Hub @DNADirectedDawn, of @EmoryGCTP, discusses severity #scoring systems as #screening for #lysosomalstoragediseases.
► 27:51 Muscle Function Assessment in Pompe Disease Pompe Disease Content Hub Jordi Diaz-Manera, MD, PhD, of @jwmdrc, discusses #muscle function and #QoL in #PompeDisease.
► 22:36 Current State of Knowledge Regarding Pompe Disease Pompe Disease Content Hub John Crowley, of @amicusrx1, discusses current knowledge regarding #PompeDisease.
► Patient Data Collected at Home Is Reliable for Assessing Progression in Pompe Disease Pompe Disease Content Hub Researchers from Duke University report that QMUS and hEIM are associated with muscle strength and function in #Pompe disease.