CLINICAL PERSPECTIVES ++++ ++++ ++++

CLINICAL PERSPECTIVES Impact of Approval of Pegcetacoplan for Rare Kidney Diseases https://checkrare.com/impact-of-approval-of-pegcetacoplan-for-rare-kidney-diseases/ Carla Nester, MD, University of Iowa Stead Family Children’s Hospital and lead investigator of the VALIANT study, discusses the impact of the recent approval of pegcetacoplan (Empaveli) for patients with C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Clinical Experience With Iptacopan for Treatment of PNH https://checkrare.com/clinical-experience-with-iptacopan-for-treatment-of-pnh/ Jamie Koprivnikar, MD, Hematologist Oncologist at Hackensack University Medical Center, discusses her clinical experience with iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Linvoseltamab in the Treatment of Relapsed/Refractory Multiple Myeloma https://checkrare.com/linvoseltamab-in-the-treatment-of-relapsed-refractory-multiple-myeloma/ Joshua Richter, MD, Associate Professor of Medicine at the Tisch Cancer Institute, Director of Multiple Myeloma at the Blavatnik Family- Chelsea Medical Center at Mount Sinai, discusses the recent U.S. Food and Drug Administration (FDA) approval of linvoseltamab for the treatment of relapsed/refractory multiple myeloma (MM). Evaluating Fenfluramine in Patients With Lennox-Gastaut Syndrome https://checkrare.com/evaluating-fenfluramine-in-patients-with-lennox-gastaut-syndrome/ Amelie Lothe, PhD, Global Medical Community Head for Rare Epilepsies at UCB, discusses recent data evaluating fenfluramine in patients with Lennox-Gastaut syndrome. Outpatient Administration of Naxitamab in Patients With Neuroblastoma https://checkrare.com/outpatient-administration-of-naxitamab-in-patients-with-neuroblastoma/ Javier Oesterheld, MD, Division Chief of the Cancer and Blood Disorders Program at Levine Children’s Hospital and Founder and Executive Director of the ARISE Cancer Consortium, discusses outpatient administration of naxitamab in patients with neuroblastoma. Immune Checkpoint Inhibition in Patients With Epstein-Barr Virus-Associated Gastric Cancer https://checkrare.com/immune-checkpoint-inhibition-in-patients-with-epstein-barr-virus-associated-gastric-cancer/ James Hamrick, MD, Chairman of the Caris Precision Oncology Alliance, discusses outcomes of immune checkpoint inhibition in patients with Epstein-Barr virus-associated gastric cancer (EBVaGC). Unmet Medical Needs in Rare Diseases https://checkrare.com/unmet-medical-needs-in-rare-diseases/ Tobias Hagedorn, Secretary of the European Society for PKU (ESPKU), discusses unmet medical needs in rare disease communities. Results From Amivanatamab Clinical Trial for Metastatic Colorectal Cancer https://checkrare.com/results-from-amivanatamab-clinical-trial-for-metastatic-colorectal-cancer/ Dirk Arnold, MD, PhD, Director of the Asklepios Tumorzentrum Hamburg, discusses results from the OrigAMI-1 clinical trial in left-sided RAS/BRAF wild-type metastatic colorectal cancer (mCRC). The ARISE Cancer Consortium https://checkrare.com/the-arise-cancer-consortium/ Javier Oesterheld, MD, Division Chief of the Cancer and Blood Disorders Program at Levine Children’s Hospital and Founder and Executive Director of the ARISE Cancer Consortium, discusses the organization’s goals and upcoming clinical trials. Risk Stratification in Patients With mIDH Glioma https://checkrare.com/risk-stratification-in-patients-with-midh-glioma/ James Hamrick, MD, Chairman of the Caris Precision Oncology Alliance, discusses age in risk stratification in patients with mutant isocitrate dehydrogenase (mIDH) glioma. Combination PD-1 Antibody and GPRC5D Antibody Therapy in Patients With Relapsed/Refractory Multiple Myeloma https://checkrare.com/combination-pd-1-antibody-and-gprc5d-antibody-therapy-in-patients-with-relapsed-refractory-multiple-myeloma/ Paula Rodríguez-Ortero, MD, PhD, Hematologist at the University of Navarra, discusses results of PD-1 inhibitor combination therapy in patients with multiple myeloma (MM). Pre-Diagnostic Symptoms and Time-to-Diagnosis in MLD https://checkrare.com/pre-diagnostic-symptoms-and-time-to-diagnosis-in-mld/ Laura Adang, MD, PhD, Assistant Professor at Perelman School of Medicine at University of Pennsylvania, discusses a cohort study conducted analyzing pre-diagnostic symptoms and time-to-diagnosis in patients with metachromatic leukodystrophy (MLD).