by Peter Ciszewski | Sep 14, 2020
Emil Kakkis, MD, President and Founder of Ultragenyx Pharmaceutical Inc., discusses the company’s mission and how the company approaches treatment development for rare diseases like Mucopolysaccharidosis VII. The goal of Ultragenyx is to develop treatments for...
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...
