Access to novel therapies for Duchenne muscular dystrophy-Insights from expert treating physicians.

Dr. Natalie Katz is currently an Assistant Professor in the Department of Pediatrics, Division of Neurology at Duke University where she co-directs the Duke Children’s Neuromuscular Program. Her team offers multidisciplinary care to children with pediatric neuromuscular diseases. She is actively involved in clinical and translational research and clinical trials with the hopes of bringing novel therapeutic treatments to children with all types of neuromuscular disease. Dr. Katz received her MD/PhD at the University of Kansas School of Medicine, and completed residency training in Child Neurology at Children’s Mercy Hospital in Kansas City, MO. She then completed a 1-year clinical neuromuscular fellowship, along with a concurrent 2-year advanced certificate program in Experimental Therapeutics at the University of Rochester in Rochester, NY. Her personal research interests are focused on understanding the natural history of children with facioscapulohumeral muscular dystrophy (FSHD), development of clinical outcomes assessments and novel methods for monitoring disease progression using imaging modalities (US and MRI) in preparation for future clinical trials in this patient population. Additional research interests include Duchenne Muscular Dystrophy (DMD), spinal muscular atrophy (SMA), limb-girdle muscular dystrophy, Charcot-Marie-Tooth (CMT), and other rare forms of neuromuscular disease.