Congenital Myotonic Dystrophy Trial Now Recruiting

The NMD4C is excited to share that an international randomized, multicenter, double-blind, placebo-controlled, phase 2/3 clinical trial testing the safety and efficacy of Tideglusib in Congenital Myotonic Dystrophy (CDM) patients aged 6-16 has begun recruiting. This is the first CDM trial to take place in Canada, even though CDM is one of the more common neuromuscular diseases in Canada, affecting more than one in 50,000 live births. The two Canadian sites are the Children’s Hospital London Health Sciences Centre (LHSC) in London, ON and the Children’s Hospital of Eastern Ontario (CHEO) in Ottawa, ON, with Drs. Craig Campbell and Hanns Lochmüller as Principal Investigators.

Both the London and Ottawa sites are open for recruitment; contact information for both sites is located below. If you would like to learn more about the eligibility criteria and for further information, please visit the ClinicalTrials.gov site to find detailed information!

LHSC site: Rhiannon Hicks, , (519)-685-8441.

CHEO site: Emilie Hill-Smith, , 613-737-7600 ext. 4014.

Our network brings together clinicians, researchers, and people with neuromuscular disease across the country.

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

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.

06-2026 NMD4C AFC Program Launch & September Workshop

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.