Today, we learned that Health Canada has granted Priority Review to Duvyzat (givinostat) for the treatment of individuals living with Duchenne muscular dystrophy (DMD).
Givinostat is a novel histone deacetylase (HDAC) inhibitor developed for Duchenne muscular dystrophy. Givinostat works differently from corticosteroids by targeting biological processes involved in inflammation, scarring, and muscle damage. By slowing these processes, givinostat is intended to help preserve muscle function and slow disease progression for individuals living with Duchenne.
Canadian individuals and families have helped advance the development of givinostat through participation in clinical trials conducted in Canada. Their time, energy, and courageous participation have helped generate the evidence supporting the therapy's development and review.
Givinostat was approved by the U.S. Food and Drug Administration in March 2024 and subsequently authorized in Europe in April 2025. More than 800 days after U.S. approval, Canadians are now seeing this therapy enter the Health Canada review process.
Priority Review is an important milestone and reflects the significant unmet need that remains for individuals and families affected by Duchenne. It may help accelerate the regulatory review timeline, but it is also a reminder that regulatory review is only one step on the pathway to access. Following a Health Canada decision, therapies must still proceed through health technology assessment, price negotiations, and provincial reimbursement processes before they become available to many Canadians.
This is exactly why Muscular Dystrophy Canada launched our What About Canada? initiative.
Too often, Canadians living with neuromuscular disorders wait years longer than individuals in other countries to access innovative therapies. For progressive conditions such as Duchenne muscular dystrophy, time matters: time is muscle. Every month of delay can mean further irreversible loss of muscle function and missed opportunities to preserve independence, mobility, participation, and quality of life.
Today's announcement is an important and encouraging milestone, but we know there is work ahead. Muscular Dystrophy Canada will continue to work in partnership with the Duchenne community, clinicians, industry partners Italfarmaco, regulators, and policymakers to help ensure that Canadians living with DMD have timely access to evidence-based treatment options, ensuring that the lived experiences of individuals and families affected by Duchenne, and the unmet needs they face, remain at the centre of regulatory, reimbursement, and policy decision-making.
Because when innovative therapies are approved and available in other countries, Canadians with Duchenne muscular dystrophy should not have to ask: What About Canada? https://t.co/3QnMVyoxa3
Leaving Montreal today after the #NMD4CSummerSchool It was an amazing workshop and networking opportunity. Thank you to all the organizers and looking forward to the amazing things to come from all the amazing colleagues and friends I made! @NMD4Canada
We're pleased to share that we have funded 9 new clinical and translational science research projects.
WATCH THE FULL ANNOUNCEMENT: https://t.co/GvzSaRiq8u
We're pleased to share that we have funded 9 new clinical and translational science research projects.
WATCH THE FULL ANNOUNCEMENT: https://t.co/GvzSaRiq8u
🎉 Ready to flex your expertise in the New Year? 💪 Join our Myogenesis Discussion Group and showcase your research! 🧬 Whether you're a seasoned pro or a budding researcher, we want to hear from you! Drop us a DM if you're interested to present in 2024! 🚨
While research is fundamental to improving human health, our environment, economy, and future, investment in research continues to be treated as a “nice to have” by successive governments.
Research requires sustained investment, especially as other countries ramp up funding.
Rest In Peace, Leslie P. Kozak: 10/28/1940 –07/13/2023. A great loss for the scientific community. Leslie's work was OFTEN years ahead of his time. His legacy lives on and his scientific contributions continue to inspire many in the field.
Les will be sorely missed. His body of work on adaptive thermogenesis was inspiring and prescient. Not to mention he laced them up for the @MapleLeafs ..legend.
It is with great sadness that we mourn the passing of Prof. Alfred Goldberg, my postdoc mentor and colleague. Fred’s contributions to science were profound and far-reaching, and his passing leaves a void. He was a passionate advocate for science and education. We will miss him.
The 2023 Ontario Exercise Physiology conference website is live! Join us in beautiful Kingston this summer. Co-hosted by Kin students+faculty from York and Queen's Universities
This meeting is for students, by students. Students present+ receive feedback from peers and faculty.
@Menzies_Lab@uOttawaBMI@OISB_IBSO@CNMDOttawa We're starting to review applications. If you are interested, please send in application now. Also, thank you to all who have RT'd!
Our lab is looking for new PhD and MSc students with a keen interest in muscle and muscle stem cell health during regeneration, disease or aging! Join us at @uOttawaBMI, @OISB_IBSO and @CNMDOttawa!
Please RT and check out our posting and website: https://t.co/Ik05iFJKQ8