24:02 Using CRISPR to Correct GAA Mutations July 17, 2021 • 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 July 17, 2021 • 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 July 17, 2021 • 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 July 17, 2021 • 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 July 17, 2021 • Pompe Disease Content Hub Mark Tarnopolsky, MD, PhD, of @McMasterU, discusses #PompeDisease #management and #research.
2:15 Newborn Screening: Communication With Parents July 17, 2021 • 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 July 16, 2021 • 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 July 16, 2021 • 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 July 15, 2021 • 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 July 12, 2021 • 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 July 1, 2021 • 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 July 1, 2021 • 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…
