by Madaline Spencer | Aug 15, 2025
Amelie Lothe, PhD, Global Medical Community Head for Rare Epilepsies at UCB, discusses recent data evaluating fenfluramine in patients with Lennox-Gastaut syndrome. Developmental and epileptic encephalopathies (DEEs) are rare, severe, lifelong epileptic...
by Madaline Spencer | Aug 14, 2025
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. ...
by Madaline Spencer | Aug 13, 2025
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). EBVaGC is a malignant tumor caused by BART miRNAs and BARF1...
by Madaline Spencer | Aug 12, 2025
Tobias Hagedorn, Secretary of the European Society for PKU (ESPKU), discusses unmet medical needs in rare disease communities. Phenylketonuria (PKU) is a genetic metabolic disorder that increases the body’s levels of phenylalanine, a natural amino...
by Madaline Spencer | Aug 11, 2025
The U.S. Food and Drug Administration (FDA) has approved Modeyso (dordaviprone) for the treatment of patients with diffuse midline glioma with a H3 K27M mutation. Glial tumors are a type of brain tumor originating in glial cells. Diffuse midline H3 K27M-mutant glioma...
