Canadian Rare Disease Network

STAGE International Speaker Seminar Series (ISSS)-Laura Arbour
2025-12-05 12:30 - 13:30 (America/Edmonton)
Reducing Genetic and Genomic Health Disparities for Indigenous People of Canada: Insights from the Silent Genomes Project

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

Webinar: 'The importance of genetic counselling in the diagnostic of rare neurological disorders' by Maria Judit Molnar
2025-12-09 07:00 - 09:00 (America/Edmonton)
Educational webinars on rare neurological, neuromuscular and movement disorders jointly organized by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN-EuroNMD) and the European Academy of Neurology (EAN).

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.

Modeling the disease to better fight it: a new approach to heart enlargement associated with RASopathies
2025-12-10 10:00 - 12:00 (America/Edmonton)
Full title: Modeling the disease to better fight it: a new approach to heart enlargement associated with RASopathies
Episode 3 of the RQMO – RARE webinar series. QC presented by Anais Medouni, PhD student in Anatomy and Cell Biology in the laboratory of Dr. Gregor Andelfinger, CHU Sainte Justine Research Center, Université de Montréal
Moderator: Jonathan Pratt, Executive Director of the RQMO

Every Relapse Matters: The Evolving NMOSD & MOGAD Treatment Landscape in Canada
2025-12-11 17:30 - 18:30 (America/Edmonton)
The Sumaira Foundation Canada is pleased to invite you to an important educational webinar on Thursday, December 11th at 7:30 PM EST. This session will explore the evolving treatment options for individuals living with AQP4+ NMOSD, seronegative NMOSD, and MOGAD in Canada.

Canadian Fragile X Research Rounds: Protein Replacement Therapy for Fragile X Syndrome
2025-12-12 12:00 - 14:00 (America/Edmonton)

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:

CMT episode 1: Challenges in the diagnosis and treatment of inherited neuropathies
2026-01-08 08:00 - 09:00 (America/Edmonton)
Delivered by Dr. Davide Pareyson (Fondazione IRCCS Istituto Neurologico C.Besta (FINCB), Milan, Italy) & Dr. Filippo Genovese (The European Charcot-Marie-Tooth Federation & ACMT-Rete per la malattia di Charcot-Marie-Tooth OdV), Thu 08 Jan 2026 at 16:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology.

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.

CMT episode 2: Progress in genetic diagnosis for neuropathies
2026-01-15 08:00 - 09:00 (America/Edmonton)
Delivered by Dr. Andrea Cortese (University College London, Department of Neuromuscular Diseases, UK), Thu 15 Jan 2026 at 16:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology.

When Nutrient Sensing Goes Wrong: How Metabolic Signals Can Drive Epilepsy and Autism
2026-01-15 10:00 - 11:00 (America/Edmonton)
4e épisode de la série de webinaires en collaboration avec Rare.qc et le RQMO.
Titre complet : When Nutrient Sensing Goes Wrong: How Metabolic Signals Can Drive Epilepsy and Autism
Paul A. Dutchak, Ph.D is an Associate Professor at Université Laval and Principal Investigator at the CERVO Brain Research Centre (Québec, Canada). His laboratory studies how genetic mutations in the GATOR1 amino acid–sensing complex drive epilepsy and autism spectrum disorders through disruptions in cellular signaling, neurometabolism, and brain excitability. His group has developed a first-in-class antisense oligonucleotide therapeutic targeting this pathway, with strong preclinical efficacy in genetic epilepsies. His research program is currently supported by the Canadian Institute of Health Research.
Moderator : Jonathan Pratt, Executive director of the RQMO

ULF Webinar: The Guardianship Transition: Minor Child to Legal Adult
2026-01-18 14:00 - 15:00 (America/Toronto)
Catherine (Catie) Becker is a Pediatric Nurse Practitioner at Massachusetts General Hospital (MGH). Catie received her master’s degree in Pediatric Acute/Chronic Advanced Practice Nursing from the University of Pennsylvania. She has worked in the MGH Leukodystrophy clinic since 2011, working with a multidisciplinary team caring for both pediatric and adult patients with leukodystrophy.

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.

CMT episode 3: Bioarkers and outcome measures for clinical trial readiness in CMT
2026-01-22 08:00 - 09:00 (America/Edmonton)
Delivered by Prof. Mary Reilly (University College London, Department of Neuromuscular Diseases, UK), Thu 22 Jan 2026 at 17:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology.

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.

CMT episode 4: Current Progress in Charcot-Marie-Tooth disease (CMT) Treatments
2026-01-29 08:00 - 09:00 (America/Edmonton)
Delivered by Prof. Kleopas Kleopa (Cyprus Institute of Neurology and Genetics (CING), Thu 29 Jan 2026 at 16:00 Paris Time This is a EURO-NMD webinar in collaboration with ERN-RND (rare neurological disorders) and European Academy of Neurology.

CATALIS-Clinical Research 101: Webinar 2
2026-01-29 18:00 - 18:30 (America/Toronto)
Webinar 2 on January 29, 2026 from 6 pm to 6:30 pm

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.

RareKids-CAN- From Evidence to Access: Advancing Pediatric Rare Disease Drug Availability in Canada
2026-02-04 15:00 - 16:00 (America/Edmonton)
Join RareKids-CAN for an in-depth discussion on the evolving role of health technology assessment and reimbursement policy in improving access to pediatric rare disease care.
