Investigator Dr. Maryam Oskoui Receives CIHR Grant to Fund Spinal Muscular Atrophy Clinical Trial
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/
Read next...
New Publication from the NMD4C Clinical Trial Network Strengthens Trial Capacity in Canada
We are pleased to share a new publication from the NMD4C Clinical Trial Network led by Kerri Lynn Schellenberg, Homira Osman, Maria Masnata, Rhiannon Hicks, Corinne Kagan, Ana Stosic, Stacey Lintern, Erin Beattie, Drs. Hanns Lochmüller, Craig Campbell, and Jean K Mah. This work focuses on the implementation of a coordinated Canadian neuromuscular clinical trial network to enhance trial readiness, capacity, and access in rare disease research.
Canadian-Led Phase II Studies Advance Vamorolone Evidence in Duchenne Muscular Dystrophy
NMD4C investigators contributed to two phase II studies evaluating vamorolone in boys with Duchenne muscular dystrophy across early childhood and adolescence, demonstrating safety, favorable pharmacokinetics, and potential efficacy. Led by Dr. Jean Mah and conducted across Canadian clinical trial sites with national and industry collaborators, the findings supported Health Canada approval of vamorolone as the first treatment for DMD in Canada. Leaders from both the clinical and patient advocacy communities, including Defeat Duchenne Canada, highlighted the impact of collaboration among patients, families, clinicians, and researchers in advancing care and improving access to new therapies.
Advancing AFC in Neuromuscular Medicine: Program Launch & September Workshop
The Royal College has approved National Training Standards for the AFC in Neuromuscular Medicine, creating a formal pathway to recognize enhanced competence with the DRCPSC credential. This designation establishes a national training standard, supports portable certification, and strengthens clinical capacity across Canada. PGME offices can now apply to offer accredited AFC programs, with a goal of five programs submitting applications by the end of 2026.