The Pompe Disease Content Hub Channel
Clinical Viewpoint on Newly Approved Combination Therapy for Pompe Disease
Pompe Disease Content Hub
In this webinar, we delve into the advancements in Pompe disease treatment, particularly focusing on the progression from mouse models to clinical trials. Key developments include the testing…
Muscular Dystrophy Association – Virtual Learning Pompe Disease
Pompe Disease Content Hub
Dr. Tahseen Mozaffar, clinical professor of neurology at the University of California Irvine, presents a webinar on Pompe Disease. Learn about the latest advancements in diagnosis, treatment options,…
Recent Videos in Pompe Disease Content Hub
Pompe Disease | Glycogen Storage Diseases
Pompe Disease Content Hub
This video dives into Pompe Disease, also known as Glycogen Storage Disease Type 2. The host explains the key components of this disease, where the body cannot break…
An Overview of Research Program for Pompe Disease
Pompe Disease Content Hub
Priya Kishnani, MD, of @DukeMedSchool, discusses #translational #research on #PompeDisease.
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.
Multidisciplinary Approach to Managing Pompe Disease
Pompe Disease Content Hub
Priya Kishnani, MD, of @DukeMedSchool, discusses #multidisciplinary care for #PompeDisease.
Newborn Screening for Pompe Disease
Pompe Disease Content Hub
Damara Ortiz, MD, of @ChildrensPgh, discusses #screening for #PompeDisease.
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.
Addressing Loneliness and Isolation in Pompe Disease
Pompe Disease Content Hub
Dr Sue Hawkins discusses how to combat #loneliness and #isolation in #PompeDisease.