by Madaline Spencer | Dec 17, 2024
Amy Dickey, MD, Assistant Professor of Medicine at Harvard Medical School, and a patient with erythropoietic porphyria (EPP), discusses the development and testing of a wearable device for light exposure measurements for patients with EPP. EEP is a rare...
by Madaline Spencer | Dec 16, 2024
Megan McLaughlin, Vice President Medicine Development Leader at GSK, discusses data from the GLISTEN phase 3 clinical trial for primary biliary cholangitis (PBC). PBC is a chronic, progressive, autoimmune, liver disease in which the bile ducts become...
by Madaline Spencer | Dec 13, 2024
Susan Dillon, PhD, CEO of Aro Biotherapeutics, discusses the initiation of a phase 1b clinical trial for late onset Pompe disease. Pompe disease is a rare genetic, lysosomal disorder that causes progressive weakness in heart and skeletal muscles. It is...
by Madaline Spencer | Dec 12, 2024
Steve Hughes, MD, Chief Medical Officer of Avidity Biosciences, discusses the initiation of a biomarker cohort in the Phase 1/2 FORTITUDE trial of delpacibart braxlosiran (del-brax) in patients with facioscapulohumeral muscular dystrophy (FSHD). FSHD is...
by Madaline Spencer | Dec 11, 2024
Courtney Olson, MD, Beth Israel Deaconess Medical Center, discusses results of a survey of patients with hereditary angioedema (HAE) and their preferences for an oral long-term prophylactic (LTP) treatment. HAE is a rare disease characterized by...
