► 1:26 Promising New Enzyme Replacement Therapy for Late-Onset Pompe Disease Pompe Disease Content Hub Priya Kishnani, MD, from @DukeMedSchool, discusses the use of #avalglucosidase alfa in late-onset #PompeDisease.
► 25:58 Pain Management in Pompe Disease Pompe Disease Content Hub Jonathan Rajan, MBChB, from the Salford Royal Foundation Trust, discusses #pain in patients with #PompeDisease.
► A Tool for Personalized Immunogenicity Risk Assessment Pompe Disease Content Hub Anne S. De Groot, MD, of EpiVax, Inc., and the Center for Vaccines and Immunology, University of Georgia, and colleagues present a study designed to develop a #tool…
► 24:02 Using CRISPR to Correct GAA Mutations Pompe Disease Content Hub Kris Saha, PhD, of @UWMadEngr, discusses #CRISPR and #genomeediting in #PompeDisease.
► 5:15 Safety and Efficacy of New Enzyme Replacement Therapy for Pompe Disease Pompe Disease Content Hub @HaniKushlaf, of @UCNeuroPMR, discusses the #safety and #efficacy of #avalglucosidase alfa in #PompeDisease.
► 38:43 Nutrition and Diet Considerations in Patients With Pompe Disease Pompe Disease Content Hub Gisela Wilcox, BMedSc, MBBS, MD, of Salford Royal NHS Foundation Trust, discusses diet and nutritional considerations in Pompe disease.
► 44:48 An Overview of Pompe Disease Pompe Disease Content Hub Stephanie Austin, MS, MA, of @DukeMedSchool, provides an overview of #PompeDisease, including #GeneticCounseling availability.
► 59:05 Diagnosis, Management, and Research Related to Pompe Disease Pompe Disease Content Hub Mark Tarnopolsky, MD, PhD, of @McMasterU, discusses #PompeDisease #management and #research.
► 2:15 Newborn Screening: Communication With Parents Pompe Disease Content Hub Dawn Laney, MS, CGC, CCRC, of the Emory Genetic Clinical Trials Center, discusses #communication with parents regarding #newbornscreening.
► 3:05 A Mother’s Perspective on Newborn Screening and Pompe Disease Pompe Disease Content Hub Melanie McKay, a mother of a child with #infantile-onset #PompeDisease, discusses the importance of #newbornscreening.
