Grant: Network Gr. in Skin Health, Bone Health, and Muscular Dyst. - Muscular Dystrophy

Neuromuscular disorders (NMDs) are rare genetic conditions that affect the skeletal muscles, nerves controlling the muscles, or junctions between muscle and nerve. They affect over 64,000 Canadians, 40% of whom are children, and result in lifelong disability, decreased wellbeing and shortened life expectancy. Research has transformed our ability to diagnose and treat NMDs, but their rarity and diversity means that networking and collaboration is essential to continued progress. In 2020, with a three-year grant from CIHR and Muscular Dystrophy Canada (MDC), we launched NMD4C, a pan-Canadian network of clinical, scientific and patient experts. We developed research resources, launched specialist training and education, provided leadership and advocacy to improve access to treatments, and engaged with patient partners to improve care, research, and collaboration. For the next phase of NMD4C, we have partnered with MDC to address emerging challenges and continue to fulfil our mission to improve NMD care and treatment for all Canadians. In this new application, we are bringing together an expanded group of patient partners, clinicians, and researchers with a research plan that will: 1) Strengthen cutting-edge preclinical research through new ways to exchange protocols and expertise and training young scientists in new technologies; 2) Increase clinical research capacity in Canada by developing a national clinical trial network and uniting local efforts in outcome measure and natural history research; 3) Improve NMD care and treatment access by developing a new community of practice, research addressing post-marketing challenges including health economics, drug access and real-world evidence, and educating NMD specialists and equipping them to apply emerging knowledge and tools. These goals will be achieved by: 4) Providing a Canada-wide platform for communication and best-practice sharing to sustain a supportive, collaborative, networked community of NMD stakeholders.