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...
by Madaline Spencer | Dec 10, 2024
Bruce Leuchter, MD, CEO of Neurvati Neurosciences and GRIN Therapeutics, discusses the initiation of the Astroscape clinical trial for tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II. TSC is characterized by the growth of...
