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 7, 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 the organization’s goals and upcoming clinical trials. The ARISE...
by Madaline Spencer | Jul 28, 2025
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). MLD is...
by Madaline Spencer | Jul 24, 2025
Jack Johnson, Co-Founder and Executive Director of FSIG, discusses symptom and treatment burden in Fabry disease. Fabry disease is a rare lysosomal storage disease characterized by a deficiency in the enzyme alpha-galactosidase (alpha-GAL). Alpha-GAL...
by Madaline Spencer | Jul 17, 2025
Ann Leon, PharmD, Global Medical Affairs Leader of Benign Hematology at Johnson & Johnson, discusses the unmet needs of patients with warm autoimmune hemolytic anemia (wAIHA). wAIHA is a rare hematologic condition characterized by the immune system...
