Pediatric Neurologist, Children’s Hospital – London Health Sciences Centre
Associate Professor, Department of Epidemiology and Clinical Neurological Sciences, Western University
Scientist, Children’s Health Research Institute.
What is the focus of your research as it relates to Duchenne muscular dystrophy?
The focus of our research is in Neuromuscular Disorders, specifically DMD, DM1 and SMA. We are involved in clinical trials and Investigator initiated natural history studies looking at clinical disease progression, quality of life and functional outcomes.
What is the most rewarding aspect of your role as a researcher and/or clinician?
Working alongside resilient children and their families who are living with muscle disorders day to day is the most rewarding and motivating part of my job.
How does the funding you receive from Jesse’s Journey make a difference to your research? What are you currently doing that you could not do without funding from Jesse’s Journey?
We have been very fortunate to have been able to work with and receive funding from Jesse’s Journey for several projects. The funding provided from Jesse’s Journey was integral in the foundation of the Canadian Neuromuscular Disease Registry (CNDR). The CNDR has now been functional since 2009. The registry is a data collection tool that allows for longitudinal collection of clinical outcomes for index neuromuscular diseases including DMD. The registry also allows patients to be informed about research studies they may qualify for. The CNDR has been used by several pharmaceutical companies, and locally we have used the CNDR for the recruitment of some of our studies. We are also the lead site for Myoblast transplantation in boys with DMD that was supported in part by Jesse’s Journey. We are s site for the STEADFAST study supported by Jesse’s Journey.
Is there anything you would like to say to Duchenne families and donors of Jesse’s Journey?
Simply thank you. The people that make up the Jesse’s Journey Family are amazing advocates for the DMD community in every way!