Canadian-Led Phase II Studies Advance Vamorolone Evidence in Duchenne Muscular Dystrophy
NMD4C investigators contributed to two recent phase II studies reporting on the safety, pharmacokinetics, and exploratory efficacy of vamorolone in boys with Duchenne muscular dystrophy (DMD) across early childhood and adolescence.
The studies, published in Neurology (Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys) and the Journal of Neurology (Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy), were led by NMD4C members Drs. Jean Mah, Hanns Lochmüller, Hernan Gonorazky, Alberto Alemán, Maryam Oskoui, Kathryn Selby, Laura McAdam, as well as Elisa Nigro, Drs. Amanda Yaworski, Anne Marie Sbrocchi, and industry collaborators (Aki Linden, Drs. Ana de Vera, Ekaterina Gresko, and Catherine Dutreix, Santhera Pharmaceuticals; and Dr. Eric Hoffman, ReveraGen BioPharma).
These trials were funded by Defeat Duchenne Canada and conducted across Canadian clinical trial sites, reflecting strong national collaboration in neuromuscular research. The findings contributed to the evidence supporting Health Canada’s approval of vamorolone as the first DMD treatment available in Canada.
As noted by Dr. Jean Mah, Lead Principal Investigator and NMD4C Investigator:
“We are grateful to our patients and families with DMD for supporting our work. These two studies extend our experience in using vamorolone to treat younger (2 to <4 yrs) and older (7 to <18 yrs) individuals with DMD. As vamorolone is the first approved treatment for DMD in Canada, we hope that it will provide families with a viable treatment option compared to other corticosteroids. We also hope that there will be other approved therapies for Canadians living with DMD in the near future.”
— Dr. Jean Mah, Lead Investigator and NMD4C Investigator
Nicola Worsfold, Executive Director of Defeat Duchenne Canada and Duchenne Mom, also highlighted the broader impact of this work:
“Health Canada’s approval of Agamree (Vamorolone) marks an important milestone for the Canadian Duchenne muscular dystrophy community and reflects years of dedication from families, clinicians, researchers, and patient advocacy organizations working together to improve outcomes and quality of life. These findings reinforce the importance of Canadian-led neuromuscular research and collaboration in advancing care, generating real-world evidence, and helping ensure Canadian families can access innovative therapies as quickly and equitably as possible. This underscores the ongoing need for continued investment in neuromuscular research, clinical trial infrastructure, and equitable access pathways so that all Canadian families can fully benefit from the therapies they helped make possible.”
— Nicola Worsfold, Executive Director of Defeat Duchenne Canada and Duchenne Mom
Together, these publications reinforce the strength of the Canadian neuromuscular clinical research community and emphasize the importance of continued collaboration to improve outcomes and access to emerging therapies for individuals living with Duchenne.
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