by Peter Ciszewski | Sep 10, 2020
Howard Rutman, MD, vice president of medical affairs at Daiichi Sankyo, Inc. talks about the two most common treatment options for tenosynovial giant cell tumors (TGCT) – surgery or systemic treatment with pexidartinib (Turalio). TGCT are a group of rare,...
by Peter Ciszewski | Sep 7, 2020
Stormy Chamberlain, PhD, Assistant Director for University of Connecticut’s Graduate Program in Genetics and Developmental Biology, describes how her lab intends to develop a short hairpin RNA (shRNA)-based gene therapy option for patients with Angelman...
by Peter Ciszewski | Sep 4, 2020
Ralf Rosskamp, M.D., Chief Medical Director at Dicerna Pharmaceuticals discusses the latest clinical data evaluating nedosiran as a treatment option for primary hyperoxaluria (PH). PH is a family of rare disorders characterized by recurrent kidney and bladder...
by Peter Ciszewski | Sep 3, 2020
Kathleen Hawker, MD, Group Medical Director at Genentech, describes the symptoms of neuromyelitis optica spectrum disease (NMOSD) and how to diagnose this rare condition. NMOSD is a rare autoimmune disorder that targets the central nervous system, particularly...
by Peter Ciszewski | Sep 2, 2020
Swati Sathe, MD, Medical Director at CHDI Management/CHDI Foundation, discusses the crucial role biomarkers play within the Enroll-HD study by facilitating earlier intervention of Huntington disease (HD). Enroll-HD, as Dr. Sathe explains, is a multicenter,...
