FOR IMMEDIATE RELEASE February 28, 2023
Toronto, Ontario – Canadians affected by facioscapulohumeral muscular dystrophy (FSHD) could face substantial delays in accessing clinical trials and any Health Canada approved life-changing treatments and therapies when they become available. Muscular Dystrophy Canada (MDC), the Canadian Neuromuscular Disease Registry (CNDR), and the Neuromuscular Disease Network for Canada (NMD4C) are joining forces to help prevent that from happening.
“Studies show that Canadians have less frequent and timely access to therapies for rare diseases, like FSHD; that very few therapies approved elsewhere in the world are even submitted to Health Canada for regulatory approval; and, that if treatment is approved there is a vast difference in who has access, across the provinces,” said Stacey Lintern, CEO Muscular Dystrophy Canada.
There are more than a dozen FSHD therapies currently in development. MDC, CNDR and NMD4C will work together to introduce a patient registry and collect information to build a Canada-wide core dataset for FSHD to speed up development, approval and access to treatments regardless of where a person lives in Canada.
“We know from our experience with spinal muscular atrophy therapies that our government requires Canadian data when reviewing a treatment. With this FSHD dataset and registry, we want to get out ahead of the treatments being developed and address the challenges and barriers to access now. We’re also hopeful more biopharmaceutical companies will establish sites, and conduct clinical trials in Canada,” added Lintern.
“The CNDR is dedicated to working in close partnership with Muscular Dystrophy Canada and NMD4C to promote opportunities to participate in cutting-edge clinical trials for patients affected by FSHD but to also collect data towards ensuring access to future therapies once approved,” said Dr Lawrence Korngut, National Principal Investigator and neurologist at the University of Calgary.
Enabling Canadians living with FSHD to register in a single national system allows them to signal their interest in participating in clinical trials, provide the essential Canadian baseline data that the regulators want to see, and be contacted when new treatments become available,” said Dr Hanns Lochmüller, NMD4C Investigator and FSHD clinical trialist.
This FSHD dataset and registry will allow for real-world evidence generation, baseline data and ultimately improve patient care by informing best practices and standards; and, lay the groundwork for the development of additional neuromuscular disorder datasets.
By working together, we can break down barriers that prevent Canadians from accessing life-changing treatments, not just for FSHD, but for all neuromuscular disorders and investigate additional opportunities that would benefit our community.
Would you like to help determine what is included in the FSHD dataset and share your Canadian experience? Join us for a virtual roundtable by emailing research@muscle.ca
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ABOUT FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
Facioscapulohumeral muscular dystrophy (FSHD) is among the most common forms of muscular dystrophy. It is caused by genetic changes that lead to damage to muscles cells. This can cause weakening and wasting of muscle in the face, shoulders, and limbs. There is no cure for FSHD at the moment, but there are more than a dozen therapies/drugs in development.
ABOUT MUSCULAR DYSTROPHY CANADA
Muscular Dystrophy Canada’s mission is to enhance the lives of those affected with neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for a cure through well-funded research. To learn more about Muscular Dystrophy Canada, please explore our website or call 1-800-567-2873.
ABOUT THE CANADIAN NEUROMUSCULAR DISEASE REGISTRY
The Canadian Neuromuscular Disease Registry (CNDR) is a Canada-wide registry of people diagnosed with a neuromuscular disease. It collects important medical information from patients across the country to improve the understanding of neuromuscular disease and accelerate the development of and access to new therapies.
ABOUT THE NEUROMUSCULAR DISEASE NETWORK FOR CANADA
The Neuromuscular Disease Network for Canada (NMD4C) is the new pan-Canadian network that brings together the country’s leading clinical, scientific, technical, and patient expertise to improve care, research, and collaboration in neuromuscular disease.
FOR MORE INFORMATION CONTACT:
Heather RiceHeather.Rice@muscle.ca
902-440-3714
