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.
1:22 The Immunological Impact of TP53 in Patients With MDS AML Content Hub @ShahramKordasti, of @KingsCollegeLon, discusses the #immunological impact of TP53 mutations on #MDS #prognosis.
3:56 The Impact of Genetics on Treatment for Patients With MDS AML Content Hub Valeria Santini, MD, University of Florence, Italy, discusses the #genomiclandscape of #MDS.
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.
3:09 VAL-1221 in the Treatment of Pompe Disease Pompe Disease Content Hub Dustin Armstrong, PhD, president of Parasail, LLC, discusses VAL-1221 for the #treatment of #PompeDisease.
1:02 Updated Data From POUT Demonstrates Maintained Benefit Oncology Robert Jones, MD, PhD, of @UofGlasgow, discusses #updates from the phase 3 #POUT study demonstrating #DFS benefit in #UTUC.
2:43 AML Patient Case: 82-Year-Old Patient Presents With Pancytopenia AML Clinical Content Hub Part 1 – Case Presentation Daniel A. Pollyea, MD, MS, presents the case of patient “L.L.” to fellow faculty members Brian A. Jonas, MD, PhD, FACP, and Gabriel…
