The Pediatric MOVE Study

Thursday, May 15

19:00 CET | 18:00 GMT | 1:00pm Et | 12:00pm CT | 11:00am MT | 10:00am PT

More than 50% of people with FSHD develop symptoms as children or teens. When treatments to slow or stop FSHD are approved, common sense suggests that it will be best to treat patients as early as possible to save healthy muscles. Yet obstacles stand in the way of reaching this common-sense goal. Most importantly, data are scarce on how symptoms evolve over time in pediatric populations. To overcome this challenge, a new, prospective natural history study, MOVE Peds, has been launched. It is similar to the ongoing MOVE (Motor Outcomes to Validate Evaluations) FSHD study, but specifically designed for children. Initial evaluations will be conducted at seven centers across the US and one in Australia. Additional sites may be added in the future. Drs. Natalie Katz and Katy de Valle will explain the design of the study, what impact it will have, and answer your questions about how to enroll your child.

Natalie Katz, MD PhD, is 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 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.

Katy de Valle, PhD, is a senior physiotherapist at The Royal Children’s Hospital and researcher at Murdoch Children’s Research Institute in Melbourne, Australia.  For the past 20 years she has worked to enhance the lives of children with neuromuscular diseases by optimizing clinical management and contributing to pharmaceutical lead clinical trials and investigator research studies. The development and validation of clinical outcome measures to evaluate function in children with early-onset FSHD was the focus of her PhD and an area she continues to work passionately in. Through her work in outcome measure validation Dr de Valle aims to ensure that the early-onset FSHD community is ready for clinical trial when they become available.