Defeat Duchenne Canada’s National Vice-President of Research, Advocacy and Education, Nicola Worsfold, attended the 27th Annual International Congress of the World Muscle Society October 11-15, 2022, in Halifax, Nova Scotia. Below is her summary for the Canadian Duchenne community:
There was a large turnout of Canadian clinicians and researchers at the 2022 World Muscle Society Conference. This is the first year the conference was held in person since the pandemic and the first time Defeat Duchenne Canada had a table highlighting our work here in Canada.
- Data was presented on the Vision DMD trial (vamorolone) and bone fracture burden. Vamorolone 6 mg/kg/day was comparable to prednisone in benefits, and the safety profile is looking positive with no stunting of growth, less bone fracture burden, and possibly fewer behavioural disturbances.
- Data on different steroid regimes for Duchenne muscular dystrophy were presented, and concluded daily steroids had better outcomes on motor function, as did the early start of steroids.
- Sarepta Therapeutics held a symposium with Professor Muntoni, Dr. Ciafaloni, and Dr. McDonald, where they presented on clinical outcomes in Duchenne and the usefulness of using external control groups instead of a placebo-controlled group in clinical trials.
- The PTC Therapeutics symposium was presented by Dr. Leanne Ward, Dr. Craig McDonald, and Dr. Eugenio Mercuri. Overall takeaways were that the new 2018 published standards of care positively impact time loss of walking, which has inadvertently improved cardiac and pulmonary outcomes. Looking at a subgroup of patients in the Ataluren clinical trials with a 6MWD (minute walk distance) between 300-400 meters showed a significantly slowed rate of decline by 30% compared to the placebo.
- Dr. Eugenio Mercuri presented Italfamaco’s Givinostat Phase 3 results which showed a continued good tolerability profile and met its primary endpoint of a faster 4-stair climb of 1.78 seconds compared to the placebo. Givinostat also reduced the decline in motor function by 40%.
- Dr. Michael Rudnicki presented his stem cell research and the early stages of his investigational therapy. Defeat Duchenne Canada continues to sponsor his research towards finding a potential treatment that targets stem cells to improve muscle regeneration in Duchenne.
Overall, Duchenne muscular dystrophy was very well represented in the poster sessions, a great testament to research progress happening in the field. If you have any questions about the science or the research presented at the World Muscle Society, please contact firstname.lastname@example.org.