EPCB Working Group Launches Patient Oriented Research Resources Tool
The NMD4C Expert Patient Capacity Building (EPCB) working group is thrilled to announce the launch of a Patient Oriented Research (POR) Resources Tool!
Curated by EPCB coordinator Patricia Mortenson with input from the entire group, this collection of online resources and websites was sourced primarily from organizations and centres across Canada, but includes some from across the world. These resources are for all stakeholders engaged in POR, including people affected by neuromuscular diseases, researchers, and advocates.
You can find the interactive tool on the NMD4C website here.
Canadians with neuromuscular diseases, their families, and advocates are increasingly asked to take an active role in research. Research and advocacy training empowers patients and ensures they have the confidence and knowledge needed to inform health care and research decisions. For researchers, patient engagement resources and training provide the tools to partner more successfully with patient-partners.
Working together, patients and researchers can achieve more.
Read more about the NMD4C’s work in training patients and research teams.
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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.