by Peter Ciszewski | Apr 4, 2022
Tomek Szczudlo, VP, Global Medical Affairs, Franchise Head, Solid Tumors at Daiichi Sankyo, gives an overview of tenosynovial giant cell tumors (TGCT). As Mr. Szczudlo explains, TGCT are a group of rare, benign tumors that involve the synovium, bursae and...
by Peter Ciszewski | Apr 4, 2022
Richard Poulin, father of a young girl with aromatic L-amino acid decarboxylase (AADC) deficiency, lists two things what he wished physicians and parents knew about the ultra-rare disease. AADC deficiency is a rare genetic disorder that affects the nervous and...
by Peter Ciszewski | Apr 1, 2022
Raymond Wang, MD, Metabolic Specialist and Director of the Multidisciplinary Lysosomal Storage Disorder Program at Children’s Hospital of Orange County, discusses RGX-111, an investigational gene therapy for mucopolysaccharidosis type I (MPS I). Data...
by James Radke, PhD | Mar 31, 2022
Terence R. Flotte, MD, Provost and Executive Deputy Chancellor of the University of Massachusetts Medical School, discusses a phase 1 clinical trial evaluating an AAV gene therapy in patients with Tay-Sachs disease. Tay-Sachs disease is a rare...
by Peter Ciszewski | Mar 29, 2022
Jerry Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, discusses the advantages that gene therapy has over enzyme replacement therapy (ERT) for genetic conditions such as Fabry disease. Fabry disease is a...
