Dr. Lindsay Alfano is a physical therapist specializing in the care and evaluation of patients with progressive neuromuscular diseases and has expertise in outcome measure development, evaluation, and administration. She is involved in numerous ongoing clinical trials analyzing and measuring outcomes in children and adults with neuromuscular disease. Dr. Alfano has developed several novel functional outcome measures including the ACTIVE system, 100 meter timed test, and Neuromuscular GRO. She has presented nationally and internationally on the study of functional outcome measures and is part of an international consortium aimed at training of outcome measures for upcoming and ongoing clinical trials. Dr. Alfano’s primary research goal is to develop and promote the optimal assessment tools to measure both cross-sectional and longitudinal differences in movement ability while minimizing the burden of testing in patients with progressive neuromuscular disorders. The purpose of her research is to guide the field, both academic and in industry, to improve the efficiency of clinical trial testing and optimize clinical trial results, as well as confidence in those results.

 

Mrs Yuriko Oda, President PADM and member of TREAT-NMD Executive Committee, Tokyo, Japan

Biography:

1980: Born in Chiba Prefecture
2002: Diagnosed with distal myopathy
2006: Gave birth naturally and became a wheelchair user for life
2008: Became a board member of PADM (Patients Association for Distal Myopathies)
2010: Received the Ainowa scholarship for people with disabilities and studied in Aarhus, Denmark for six months
2014: Launched a YouTube channel called “Wheelchair Walker”
2015: PADM received the Google Impact Challenge Grand Prix
2016: Received the Youth Leader Award
2017: Created and released a user-generated barrier-free map application called “WheeLog!”
2018: Received the Innovative Practice Award awarded by Zero Project
2018: Received Second Prize at the Tokyo Open Data Application Contest
2018: Established “WheeLog” as a general incorporated corporation, appointed as CEO and founder
2018: Became a member of Town Development Meeting Committee of Tokyo Metropolitan Welfare
2019: Became a member of a project organized by the Ministry of Land, Infrastructure, Transport and Tourism which focuses on developing methods to obtain tourist-friendly barrier-free information
2019: Became a “Global Champion” in the category of XYZ at the World Summit Award

Dr Charles Gersbach, Associate Professor, Duke University, Durham NC, United States

Dr. Charles A. Gersbach is the Rooney Family Associate Professor at Duke University in the Departments of Biomedical Engineering and Surgery, an Investigator in the Duke Center for Genomic and Computational Biology, and Director of the Duke Center for Biomolecular and Tissue Engineering.  His research interests are in genome and epigenome editing, gene therapy, regenerative medicine, biomolecular and cellular engineering, synthetic biology, and genomics.  Dr. Gersbach’s work has been recognized through awards including the NIH Director’s New Innovator Award, the NSF CAREER Award, the Outstanding New Investigator Award from the American Society of Gene and Cell Therapy, the Allen Distinguished Investigator award, and induction as a Fellow of the American Institute for Medical and Biological Engineering.

 

Dr. Craig Campbell is the Deputy Chair of Pediatrics (Research), the Head of the Division of Pediatric Neurology and the medical director of the multidisciplinary neuromuscular clinic based at Thames Valley Children’s Centre and the Pediatric Neurophysiology Laboratory at Children’s Hospital London Health Sciences Centre.  He is an Associate Professor in Pediatrics, Clinical Neurological Sciences and Epidemiology at Western University and a Scientist at the Children’s Health Research Institute. His training in Pediatrics and Neurology and Epidemiology was completed at the University of Ottawa.

The pediatric NM service at LHSC/TVCC has evolved into an internationally recognized site for clinical care delivery, research and clinical outcomes evaluation.  Clinical trial work in DMD patients has been an area of academic activity, and Dr. Campbell is collaboratively involved in many academic and industry initiated clinical trial activities in pediatric neuromuscular disease. He is a member of the Muscle Study Group, World Muscle Society, and co-chair of the TREAT-NMD Registry Oversight Committee and Task Force. Dr. Campbell has been awarded the George Karpati Award Researcher of the Year in 2011 and 2015, Muscular Dystrophy Canada.

The following are specific areas of innovation:

Congenital Myotonic Dystrophy. The study of CDM has been an interest at a population level and in understanding patient reported outcome measures. We are currently involved in following a cohort of children with CDM who were identified in an active national surveillance program. We have published disease incidence from the surveillance, and compared registry methodology in this population. We are now involved in outcome measurement validation in CDM patient in anticipation of clinical trials.

Development of Canadian Neuromuscular Disease Registry I have also been instrumental in the initiation of the Canadian Neuromuscular Disease Registry as the Pediatric Lead investigator and the past head and founder of the Canadian Pediatric Neuromuscular Group. The CNDR has taken a novel approach to rare disease registry methodology and is now nearing 3000 patients enrolled across Canada. The CNDR data feeds into a global NM disease registry effort coordinated by TREAT-NMD.

Study of Quality of Life in Duchenne Muscular Dystrophy. This more recent line of research has been focused on understanding QOL, HRQOL and fatigue in DMD patients. Our work is the first longitudinal study of QOL using a large patient registry and to approach understanding of QOL using a multivariate approach. Fatigue is emerging as an important factor that impacts QOL and HRQOL and our group is one of the first to explore the role of fatigue in the DMD as an important outcome measure and a potentially modifiable factor to improve patient care.

Founder of the Canadian Pediatric Neuromuscular Group. This academic group, which includes all major pediatric NM centres across Canada, has worked together to understand NM care patterns in Canada and work on collaborative research efforts. The CPNG now functions as an academic network and promoting NM issues in Canada.