Canadian Rare Disease Network

Hope on the Horizon: Trials, Treatments and Therapies for DMD, DM1 & MG
2025-11-26 18:00 - 2025-11-28 19:00 (America/Toronto)
Muscular Dystrophy Canada in partnership with the Neuromuscular Disease Network for Canada (NMD4C)’s Clinical Trial Network is excited to host three Hope on the Horizon events!
These webinars are geared towards family members and people living with NMDs.
Join us from November 26th – 29th to learn more about the treatment and clinical trial landscape for Duchenne Muscular Dystrophy, Myotonic Dystrophy Type 1 and Myasthenia Gravis from key experts in the field!

Child Health and Wellness Seminar: The Evolution of Medical AI: Progress, Potential, and Responsibility
2025-12-01 08:30 - 09:30 (America/Edmonton)
The Evolution of Medical AI: Progress, Potential, and Responsibility Speaker: Dr. Eman Rezk, PhD NSERC Postdoctoral Fellow Department of Electrical and Computer Engineering, University of Waterloo Host: Dr. Mohamed Eltorki, MD, FRCPC Associate Professor, University of Calgary

Can-Child Webinar: F-words Foundations Workshop
2025-12-04 09:30 - 12:30 (America/Toronto)
Interactive 3-hour F-words Foundations Workshop to explore how to bring the F-words for Child Development—Functioning, Family, Fitness, Fun, Friends, and Future—into everyday practice and life!

CARELeuko Symposium 2025
2025-12-05 08:00 - 17:00 (America/Toronto)
The Canadian Association for Research Excellence in Leukodystrophy (CARELeuko) is excited to welcome you to the 2025 CARELeuko Symposium: Uniting Canada’s Leukodystrophy Community to Drive Collaboration and Accelerate Research and Care!
The 2025 CARELeuko Symposium is taking place on Friday, December 5, 2025, at the Research Institute of the McGill University Health Centre in Montreal, Quebec, Canada.

36th International Symposium on ALS/MND
2025-12-05 08:00 - 2025-12-07 17:00 (America/Los_Angeles)
The International Symposium is the biggest annual conference dedicated to ALS and MND research. Each year they bring together researchers from around the world to share new understanding of amyotrophic lateral sclerosis and motor neurone disease.

FA Together: Learn. Share. Connect. mini-regional conference in Calgary
2025-12-08 17:00 - 19:00 (America/Edmonton)

2nd International Conference on Clinical Research Networks (CRNs) for Rare Diseases
2025-12-09 08:00 - 2025-12-10 17:00 (America/Edmonton)
This second edition will convene a wide range of international experts, including researchers, clinicians, patient advocates, industry representatives, policymakers, and funders to strengthen the collaborative ecosystem that underpins clinical research in rare diseases worldwide.

Duchenne Patient Academy 2025
2025-12-13 08:00 - 2025-12-14 17:00 (America/Edmonton)

SMA Together: Learn. Share. Connect. A Mini-Regional Conference - Toronto
2025-12-13 11:00 - 14:00 (America/Toronto)
The SMA Together: Learn. Share. Connect. Mini-regional conference in Toronto is less than three weeks away! Join them, in-person on December 13 to learn about the latest updates in spinal muscular atrophy, including:

ERDERA: Joint Transnational Call 2026
2025-12-16 15:00 - 17:00 (America/Edmonton)
This information webinar on the 2026 Joint Transnational Call for Proposals is scheduled for 16 December 2025, 15:00–17:00 CET, focusing on resolving unsolved rare genetic and non‑genetic diseases.

RealiseD Webinar 1: Realising Clinical trials in Ultra-Rare diseases
2026-01-13 17:00 - 18:30 (America/Edmonton)
Join RealiseD’s first webinar to learn how RealiseD is pioneering a collaborative and comprehensive approach to clinical trials in ultra-rare diseases. During the webinar, they’ll walk you through the project’s objectives, structure, and methodologies—and show how you can be part of this transformative journey.

CanPKU+: Advocacy in Action Update Sessions
2026-01-14 19:00 - 20:00 (America/Edmonton)
Join the Advocacy in Action Update Sessions with John Adams in order to hear updates on various campaigns and what needs to be done to move things forward for the PKU community.

Rare Disease Internation-Moving the WHA Resolution on Rare Diseases Forward: From Promise to Action
2026-01-15 05:30 - 06:30 (America/Edmonton)
Join RDI for this public webinar where they will discuss the current status of the WHA Resolution implementation, as they approach the World Health Organization Executive Board meeting taking place in February 2026. This webinar will engage in a multistakeholder dialogue focusing on the necessary path forward to accelerate the implementation of the Resolution and importantly the development of the GAPRD.

Knowledge Sharing Sessions – Feasibility of a Home-Based Exergaming Intervention for Youth With Spinal Muscular Atrophy.
2026-01-19 19:00 - 20:00 (America/Toronto)
Dr. Geoff Frost will be leading NMD4C’s first Physiatrist Community of Practice (CoP) Knowledge Sharing Session. He will present a recent article authored by Iraqi et al. on a work conducted in Canada by fellow NMD4C members: “Feasibility of a Home-Based Exergaming Intervention for Youth With Spinal Muscular Atrophy.”
The session will take place on January 19th, 2026 at 7:00 PM EST. To register, please contact CoP manager Maria Masnata. Once registered, Maria will send a calendar invite so you can easily save the date. We are looking forward to seeing you there!

RealiseD Webinar 2: Evidence Assessment Framework. A need to change mindset from developers, regulators and HTA
2026-01-20 17:00 - 18:30 (America/Edmonton)
The second webinar turns the spotlight on one of the most critical issues in rare disease research: the generation and assessment of evidence. François Meyer, a leading consultant in health technology assessment, will examine why current frameworks often fail to deliver predictable and aligned outcomes, resulting in delays and inequalities in patient access. The session will explore the global and European initiatives aimed at addressing these shortcomings and present the RealiseD consortium’s efforts to advance scientific and operational solutions. More importantly, it will advocate for a fundamental shift in mindset—one that embraces flexibility and innovation in the way evidence is generated, interpreted, and assessed.

ERN-EuroBloodNet-Session 1: Strong at Any Age – Redefining Frailty Together
2026-01-22 17:30 - 18:30 (Europe/Belgrade)
EuroBloodNet Topic on Focus: Ageing with a Bleeding Disorder”!
This educational program is coordinated by ERN-EuroBloodNet together with the umbrella patient organisation EHC – European Haemophilia Consortium, represented by ePAG advocate Marius Tanase.

Healthy Living and Aortopathy
2026-01-22 19:00 - 20:00 (America/Toronto)
Save the date for your upcoming webinar with Dr. Nathaniel Moulson, focused on exercise. This webinar will take place on January 22, 2026 at 7:00pm, registration details to be released in the coming weeks.

PiPER Community of Practice for 2026
2026-01-28 12:00 - 13:00 (America/Toronto)
Save the date on *January 28th 2026* from 12-1 pm for a dynamic session titled “Lessons Learned by a Research Team New to Engaging PLEX in Randomized Controlled Trials ”.

Canada's Drug Agency-Preconsultation Webinar: Improvements to the Drug Reimbursement Review Process
2026-01-28 14:00 - 15:30 (America/Toronto)
Join CDA’s preconsultation webinar on proposed improvements and upcoming procedural changes to our drug reimbursement review process.
You will hear from their senior leaders how these plans will advance our efforts to modernize and streamline drug reimbursement reviews.

Defeat Duchenne Canada-Research Grant Announcement 2026
2026-01-29 18:00 - 19:30 (America/Toronto)
Mark your calendar for Thursday, January 29, 2026, from 6:00–7:30 p.m. ET, and join Defeat Duchenne Canada online for an evening celebrating the incredible progress of their community and the promise of the future.

Sickle Cell Awareness Group of Ontario (SCAGO) Learning for Life - Clinical Trials 101 What Every Patient Should Know Before Enrolling
2026-01-31 11:00 - 12:30 (America/Edmonton)
Considering a clinical trial? Join SCAGO for an informative virtual session designed to help patients and caregivers understand the process, benefits, and important considerations before enrolling.

RQMO: 1st Zebinary of the Month of Zebra 2026
2026-02-03 10:00 - 11:00 (America/Edmonton)
1st Zebra of the Month of Zebrier 2026 of the RQMO
Speakers:
– Dr. Roberta, La Piana, Assistant Professor, Department of Neurology and Neurosurgery, McGill University
– Dr. Thomas Durcan, Associate Professor, Department of Neurology and Neurosurgery, McGill University

RealiseD Webinar 3: Single arm, RCT or something in between – how to enrich clinical trial design and analysis in rare diseases
2026-02-03 17:00 - 18:30 (America/Edmonton)
Single-arm trials remain a cornerstone of rare disease research, but their limitations and complexities cannot be ignored. In the third webinar, experts Yulia Dyachkova (Merck Healthcare), Jenny Devenport (Roche), Cornelia Dunger-Baldauf (Novartis) abd Guillaume Canaud will lead a deep dive into the evolving role of SATs in drug development. The discussion will cover regulatory and HTA perspectives, review case studies of success and failure, and explore advanced statistical methods designed to optimise trial design. This session is not just about understanding the current landscape—it is about shaping the future of evidence generation for rare and ultra-rare diseases.

D2R-Webinar | Anti-Black Racism in Health Research
2026-02-04 12:00 - 13:00 (America/Edmonton)
In honour of Black History Month, D2R invites you to an interactive webinar on “Anti-Black Racism in Health Research.” This session will explore the history of slavery in Canada, trace the evolution of anti-Black racism within Canadian health research, and highlight practices and strategies to address and counter these challenges.

RQMO: 2nd Zebinary of the Month of Zebra 2026
2026-02-05 10:00 - 12:00 (America/Edmonton)
Speaker: Dr. André Megarbane, Professor, Department of Human Genetics, lebanese American University, Beirut, Lebanon

OSSU World Café Conversations Series:"Find and Seek: Conversations on Matching Researchers and Lived Experience Partners"
2026-02-06 10:00 - 12:00 (America/Edmonton)
OSSU World Café Conversations series with Maureen Smith🇨🇦, Aleisha F. and supported by Ontario SPOR SUPPORT Unit (OSSU):
“Find and Seek: Conversations on Matching Researchers and Lived Experience Partners”

RQMO: 3rd Zebinary of the Month of Zebra 2026
2026-02-09 10:00 - 12:00 (America/Edmonton)
3rd Zebinary of the Month of Zebrier 2026
Speaker: – David Cohen, Professor, Faculty of Health, Sorbonne University, Paris, France

8th TREAT-NMD® International Conference – Lisbon, 2026
2026-02-10 08:00 - 2026-02-12 17:00 (Europe/Lisbon)
Join TREAT-NMD at this vital, in-person gathering where patients, clinicians, researchers, industry, and regulators come together to tackle the biggest challenges in neuromuscular treatment development, diagnosis and care. Topics will include; disrupted clinical trial delivery, regulatory uncertainty, global access inequalities and shrinking public funding, they’ll tackle these themes with a holistic, solutions-driven approach and a patient centric and global focus.

RealiseD Webinar 4: Enhancing patient-centricity in Rare Disease Clinical Trials
2026-02-10 17:00 - 18:30 (America/Edmonton)
The final webinar focuses on what truly matters: the patient. Rare disease trials often struggle to capture outcomes that reflect patient priorities but RealiseD is working to change that. Rudradev Sengupta, Senior Trial Design Lead at One2Treat, will present an approach that embeds patient voices into every stage of trial design and analysis. By using hierarchical endpoints and net treatment benefit, this framework balances benefits and risks in alignment with patient preferences, enabling transparent discussions among stakeholders and ensuring that success is defined by what matters most to patients. This is a vital conversation for anyone committed to making clinical research more humane and impactful.

RQMO: 4th Zebinary of the Month of Zebra 2026
2026-02-11 10:00 - 12:00 (America/Edmonton)
Speaker: – Jacques P. Tremblay, Ph.D, Full Professor, Department of Molecular Medicine, Faculty of Medicine, Université Laval
