Together with Muscular Dystrophy Canada (MDC) we are pleased to invite you to a webinar on therapies for Duchenne muscular dystrophy. This webinar is primarily targeted at Canadian clinicians, academics, and trainees with an interest in neuromuscular disease. This is an independent clinical/academic webinar with no industry sponsorship or influence, and NMD4C and MDC are providing organizational and technical support.

Chair

• Dr. Hanns Lochmüller | Senior Scientist, CHEO Research Institute, Professor of Neurology, University of Ottawa Faculty of Medicine and The Ottawa Hospital Department of Medicine

Agenda

Mobilizing muscle stem cells as a treatment for DMD

• Dr. Michael Rudnicki | Professor of Medicine and Cellular and Molecular Medicine, and Program Director of Regenerative Medicine at uOttawa, Scientific Director of the Canadian Stem Cell Network, Senior Scientist and Director of the Regenerative Medicine Program and the Sprott Centre for Stem Cell Research at Ottawa Hospital Research Institute

Vamorolone, a novel “dissociative” steroid for the treatment of Duchenne Muscular Dystrophy: Can We Separate the Good from the Bad?

• Dr. Leanne Ward | Professor of Pediatrics and Tier 1 Clinical Research Chair in Pediatric Bone Disorders at uOttawa, Medical Director of the CHEO Genetic and Metabolic Bone Disease Clinic, Scientific Director of the Ottawa Pediatric Bone Health Research Group, Pediatric Endocrinologist at CHEO

Learning Objectives

1. Understanding the muscle stem cell deficit in DMD.
2. Development of small drugs that enhance muscle regeneration by mobilizing muscle stem cells.
3. To review the benefits and challenges of classic glucocorticoid therapy (prednisone, deflazacort) for the treatment of DMD
4. To discuss VBP15 (vamorolone), a novel “dissociative steroid” developed with the goal to separate the anti-inflammatory benefits of steroid therapy from the well-known adverse effects
5. To summarize the results of clinical trials in ambulatory boys with DMD that highlight the impact of vamorolone on muscle, bone and endocrine outcomes

The NMD Rounds & Educational Webinars is a self-approved group learning activity (Section 1) as defined by the Maintenance of Certification Program of the Royal College of Physicians and Surgeons of Canada.