NMD4C: The Neuromuscular Disease Network for Canada

NMD4C investigator Dr. Maryam Oskoui has been awarded a Canadian Institutes of Health Research Clinical Trials Operating Grant ($1,777,095) for INFORM SMA as nominated principal applicant. Several NMD4C members are listed on this grant as co-investigators, including Katrina Cardiff, Shaainthabie Karthigesu, Dr. Kathryn Selby, Dr. Jean Mah, Dr. Heran Gonorazky, Dr. Hugh McMillan, Dr. Jordan Sheriko, Dr. Victoria Hodgkinson, and Dr. Lawrence Korngut; and as collaborators, including Niamh Cushen, Catherine Desautels, Dr. James Dowling, Monique Emond, Stacey Lintern, Sarah Turgeon-Desilets, Susi Vander Wyk, and Angelina Woof.

INFORM SMA

INFORM SMA is embedded in the INFORM RARE network, whose vision is to improve health care for children with rare genetic diseases by generating real-world evidence to support decision-making about new and existing therapies. The goal of this trial is to evaluate the effectiveness of a home-based exergame intervention in promoting physical activity for children and youth with Spinal muscular atrophy (SMA) while validating digital outcome measures. Multiple knowledge users (patients, parents, healthcare providers, and researchers) have informed the design of both the exergame intervention and the trial. This registry-based trial will also identify the best tools to measure meaningful change in this population and provide the framework for future pragmatic trials in SMA.

CIHR recently featured Dr. Oskoui’s innovative trial in their “Faces of Health Research” initiative. Read the full feature to learn more!

Links

1. https://cihr-irsc.gc.ca/e/53357.html?filter=Maryam+Oskoui
2. https://www.informrare.ca/