New Publication Guidance on gene replacement therapy in Spinal Muscular Atrophy: a Canadian perspective
The NMD4C is proud to announce that a Guidance on gene replacement therapy in Spinal Muscular Atrophy: a Canadian perspective has been accepted for publication by the Canadian Journal of Neurological Sciences. Notably this consensus statement is authored by NMD4C members Drs. Maryam Oskoui, Hernan Gonorazky, Hugh McMillan, James Dowling, Reshma Amin, Cynthia Gagnon, and Kathryn Selby.
This publication is the first significant research output of the NMD4C, and is a product of the network’s development, dissemination, and implementation of clinical guidelines working group. The working group invited pediatric neuromuscular clinical experts from across the country to provide feedback and direction in adapting existing consensus statements on gene replacement therapy in SMA to the Canadian context.
“It is an exciting time for the neuromuscular field with the advancement of gene therapies. It is our duty as neuromuscular specialists to advocate for safe, equitable, and transparent access to these treatments for all Canadians. We hope that through this consensus we can help inform the application, indication and use of gene replacement therapy in Spinal muscular atrophy.”
– Dr. Hernan Gonorazky.
You can find the publication here.
With three SMA therapies now commercially available in Canada, the working group set out to provide guidance by adapting a set of European consensus statements to a Canadian context and highlighting areas of future need for gene replacement therapy in SMA in Canada. The original publication from an ad-hoc group of 13 European SMA experts who published a set of consensus statements on the use of gene replacement therapy in SMA using a Delphi consensus process.
The NMD4C will continue to work towards its knowledge translation (KT) goals to improve care standards, train the next generation of neuromuscular researchers and clinicians, and strengthen research infrastructure for better research and more treatments.
Read next...
You Can Now Find NMD4C on Bluesky
We’re moving to Bluesky! The NMD4C will now share updates on Bluesky instead of X. Please consider following us at @nmd4c.ca to stay connected!
CIHR Funds Interdisciplinary Project to Study SBMA in Indigenous Peoples of the Prairie Provinces
The NMD4C is excited to share that network investigators have been awarded funding from the CIHR for an Interdisciplinary Spinal-bulbar muscular atrophy study in Indigenous peoples of the prairie provinces, addressing the community needs, and developing clinical knowledge and therapies.
NMD4C Presents the 2025 Early Career Award Recipients
We are excited to announce the recipients of the 2025 NMD4C Early Career Awards! This is the third year of our annual award program, celebrating excellence and contribution to the neuromuscular field from early career investigators across both clinical and basic science streams.
Muscular Dystrophy Canada Launches Initiative to Improve Genetic Testing Access for Myotonic Dystrophy
Muscular Dystrophy Canada is thrilled to launch an innovative initiative designed to tackle the diagnostic hurdles experienced by individuals with myotonic dystrophy.
New Content Available in LEARNMD
LEARNMD is the NMD4C’s online educational platform, offering talks and sessions on a wide range of neuromuscular topics presented by leading clinical and scientific experts worldwide. We’re excited to share what’s new on LEARNMD in February 2025!
Quebec Network Réseau Québécoise de l’Amyotrophie Spinale Joins NMD4C as Community of Practice Sub-Group
We are thrilled to announce that the RQAS, a network uniting rehabilitation clinicians across Quebec to advance the care of people with Spinal Muscular Atrophy has officially become a sub-group Community of Practice of the NMD4C