by Madaline Spencer | Mar 24, 2025
Alessandra Maleddu, MD, Sarcoma Specialist at the University of Colorado, discusses the misdiagnosis of desmoid tumors and provides some resources for patients. Desmoid tumors are rare, benign, locally invasive, soft tissue tumors. They are commonly...
by Madaline Spencer | Mar 20, 2025
Bruce Leuchter, MD, Co-founder and CEO of Neurvati Neurosciences and GRIN Therapeutics, discusses the Breakthrough Therapy designation for radiprodil for treatment of patients with GRIN-related neurodevelopmental disorders. GRIN-related...
by Madaline Spencer | Mar 19, 2025
Steven Pipe, MD, Professor of Pediatrics and Pathology at the University of Michigan, discusses positive follow-up results in patients with hemophilia B treated with Hemgenix (etranacogene dezaparvovec-drlb). Hemophilia B is a rare bleeding disorder that...
by Madaline Spencer | Mar 18, 2025
Alan Percy, MD, Pediatric Neurologist at the University of Alabama at Birmingham, discusses results from the DAFFODIL study evaluating long-term safety of trofinetide in girls ages two to four years with Rett syndrome. Rett syndrome is a...
by Madaline Spencer | Mar 17, 2025
Reena Sharma, MD, Adult Metabolic Consultant at Salford Royal Hospital and University of Manchester, discusses plans for a phase 3 clinical trial evaluating FLT201 in patients with Gaucher disease. Gaucher disease refers to a rare group of inherited metabolic...
