Overview
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 research and multiple 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 characterizing children with facioscapulohumeral muscular dystrophy (FSHD) 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.
Current Appointments & Affiliations
Recent Publications
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.
Journal Article Neuromuscul Disord · August 23, 2025 Gene transfer therapy represents a major advancement in the treatment of patients with Duchenne muscular dystrophy (DMD). As clinical use expands, there is an urgent need for standardized, evidence and practice-informed guidelines to ensure safe and equita ... Full text Link to item CiteReductions in functional muscle mass and ability to ambulate in Duchenne muscular dystrophy from ages 4 to 24 years.
Journal Article J Physiol · October 2024 Duchenne muscular dystrophy (DMD) results in a progressive loss of functional skeletal muscle mass (MM) and replacement with fibrofatty tissue. Accurate evaluation of MM in DMD patients has not previously been available. Our objective was to measure MM usi ... Full text Link to item CiteReply: Wheelchair use in genetically confirmed FSHD1 from a large cohort study in Chinese population.
Journal Article Brain · June 30, 2022 Full text Link to item CiteRecent Grants
A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants with Duchenne Muscular
Clinical TrialPrincipal Investigator · Awarded by Sarepta Therapeutics, Inc. · 2025 - 2035MOVE+ Peds (Fixed rate)
ResearchPrincipal Investigator · Awarded by University of Kansas · 2025 - 2029MOVE+ Peds
ResearchPrincipal Investigator · Awarded by University of Kansas · 2025 - 2029View All Grants