Canadian Rare Disease Network

Mito Med 2025 Masterclass on Primary Mitochondrial Myopathy with a Focus on TK2d
2025-06-21 08:00 - 17:00 (America/Kentucky/Louisville)
New this year for Clinical-Scientific Attendees to UMDF’s #MitoMed Conference — a Masterclass on Primary Mitochondrial Myopathy (#PMM) with a Focus on #TK2d. Tracks for Early Career Investigators and for Up-and-Coming Clinicians.

Defeat Duchenne Canada-Industry Partner Update: REGENXBIO
2025-06-23 16:00 - 17:00 (America/Edmonton)
Join Defeat Duchenne Canada for an educational webinar featuring Dr. Jahannaz Dastgir, Executive Director of Clinical Development at REGENXBIO, as she shares the latest updates on RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, and their Phase 1/2/3 AFFINITY DUCHENNE® trial.
This session will provide valuable insights into the science behind RGX-202, trial progress, and what this potential investigational treatment could mean for the Duchenne community. Whether you are a parent, caregiver, clinician, or advocate, this webinar is an opportunity to stay informed and ask questions directly to the team leading this important research.

NMD Rounds & Educational Webinars-Bulbar impact of SMA: approach to assessment and treatment
2025-07-08 17:00 - 18:00 (America/Toronto)
NMD4C and MDC are pleased to invite you to a CPD-accredited webinar Bulbar impact of SMA: approach to assessment and treatment. NMD4C and MDC are providing organizational and technical support. This webinar is supported by Novartis through a restricted educational sponsorship with no involvement or influence on program content.

Rare Disease Day 2026 - Concept Introduction
2025-07-21 07:00 - 08:00 (America/Edmonton)
Company representatives and Rare Disease Day National Partners are warmly invited to this introductory session unveiling the vision for the 2026 campaign.

IMPaCT Webinar - An Overview of Current and Future Clinical Trials in Children: a European Perspective
2025-07-21 12:00 - 13:00 (America/Toronto)
Dr. Eva Degraeuwe (MD-PhD) is a dedicated pediatric resident and researcher in pediatric nephrology and global clinical trial optimilisation. Working in many Global networks focused on rare and low prevalance diseases, she has completed her PhD in 2024 participating and guiding over 35 clinical trials. Moreover, she has a passion for patient engagement, especially within a clinical trial setting.
Webinar Overview:
– European network setting and the role of networks
– Pathways to clinical trial optimilisation
– Patient engagement and what has been achieved

Community of Practice- Beyond Inclusion: Co-Designing with Lived Experience to Build Meaningful Partnerships in Stroke Research
2025-07-30 12:00 - 13:00 (America/Edmonton)
📢 On July 30th from 12:00 – 1:00 pm EST Emine Kocabas, Ashley Berger, & Vishma Sookdeo will be speaking about “Beyond Inclusion: Co-Designing with Lived Experience to Build Meaningful Partnerships in Stroke Research”.

ALS Canada: Driving & ALS Webinar
2025-08-13 13:00 - 15:00 (America/Edmonton)
Join ALS Canada and Sunnybrook Health Sciences Centre to learn about the impact of ALS on driving ability, adaptive driving equipment, how an ALS diagnosis may affect insurance, and available alternative options.

PxP Africa 2025: Virtual Meeting August 13, 2025 From Research Participants to Patient Partners in Research: Centering African Patients Voices
2025-08-13 18:00 - 20:00 (America/Edmonton)
PxP Africa is the first regional PxP meeting, led by Joab Wako and Kwanele Asante who are members of the 2025 steering committee for the global PxP conference taking place in September. PxP Africa will spotlight the critical role of patient partners—especially African patients—as active contributors in research, not just research participants. The session will challenge the traditional top-down model of research and explore how lived experience can inform, shape, and lead meaningful, inclusive studies.

PxP Australia 2025
2025-08-19 08:00 - 09:00 (America/Edmonton)
PxP Australia is a regional webinar, led by Letisha Living and Janelle Bowden who are members of the 2025 steering committee for the global PxP conference taking place in September. The theme of PxP Australia is “working well together” and Dr. Joan Carlini will be joining the session as a speaker.

Genome Editing in Stem Cells and Regenerative Medicine
2025-08-19 12:00 - 13:30 (America/Edmonton)
CRISPR-based genome editing has revolutionized biomedical research, offering powerful tools to investigate gene function, model diseases, and develop novel therapies. This focused workshop will provide participants with both theoretical grounding and practical insights into current genome editing technologies.
The Stem Cell Network (SCN) is pleased to offer the online workshop “Genome Editing in Stem Cells and Regenerative Medicine”. This workshop will equip participants with both a theoretical grounding and practical insights into current genome editing technologies. It will also explore key concepts in experimental design, delivery, and selection methods.

Webinar: Patient Partnership 2025 – The Lupus100 Project
2025-08-27 00:00 (America/Edmonton)
Co-developed by ERN ReCONNET and LUPUS EUROPE, The Lupus100 Project is a multilingual resource based on real patient questions about lupus, turning a national patient booklet into a Europe-wide, medically validated information tool. Speakers Eric Hachulla (clinician, ERN ReCONNET), Jeanette Andersen (patient representative, ERN ReCONNET), and Zoe Karakikla-Mitsakou (General Secretary, Lupus Europe) will share how meaningful collaboration between patients and clinicians shaped this impactful communication initiative!

WHA Resolution Coalition Webinar: Turning the WHA Resolution on Rare Diseases into Action
2025-08-28 06:00 - 07:30 (America/Edmonton)
This Coalition webinar will further discuss the formalization of the Coalition as well as the updates and next steps in the implementation of the WHA Resolution on Rare Diseases and the development of the Global Action Plan.

Back-to-School with PH: Q&A Session
2025-09-04 11:00 - 12:00 (America/Edmonton)
Getting ready for a new school year can be exciting—but also challenging when pulmonary hypertension (PH) is part of the picture.
Join in on Thursday, Sept 4 at 11:00 AM EST for a virtual round table with Janette Reyes, PH Pediatric Nurse at SickKids Hospital.

IMPaCT Webinar: Lessons Learned from the Innovative Pediatric Clinical Trials (iPCT) Network
2025-09-10 10:00 - 11:00 (America/Edmonton)
Speakers: Tannis Erickson & Naveen Poonai Title: Lessons Learned from the Innovative Pediatric Clinical Trials (iPCT) Network

RareKids-CAN-Education Session: Family Engagement in Research Fundamentals
2025-09-17 14:00 - 15:00 (America/Toronto)
Join RareKids-CAN on September 17, 2025, from 2-3pm ET for their next education session, led by their Patient and Family Engagement Facilitators. This session is open to everyone interested in strengthening engagement practices. Don’t miss out!

EURORDIS Webinar: Patient Partnership 2025 – The Lupus100 Project
2025-09-18 18:00 - 19:00 (America/Edmonton)
The Lupus100 Project, recognized as a winner of the 2025 Patient Partnership Good Practice Challenge, highlights the crucial role of patient partnership in improving communication about rare diseases such as lupus.

LHON Awareness Day 2025 Webinar: "Exploring the Power of Nutrition in Mitochondrial Diseases"
2025-09-19 15:00 - 17:00 (America/Edmonton)
This year’s theme focuses on how nutrition can support mitochondrial health. You’ll hear from experts across Australia, Italy, and Brazil, sharing practical strategies and the latest research relevant to LHON and other types of mito.

Advancing Rare Disease Healthcare: Spotlighting the Economic and Health System Burden of Disease
2025-09-23 09:30 - 11:30 (America/Edmonton)
Please join this 1.5-hour live virtual meeting to hear esteemed faculty describe the global economic impact of rare diseases on patients and healthcare systems. Expert community advocates will also share their practical experience of advancing patients’ needs with key stakeholders. Panel discussion and audience questions will consider how to have meaningful impact in your country or region with patient healthcare experience data and insights to drive change in practice and better health outcomes for people living with rare disease. The live virtual meeting is further supplemented with published studies and content.

La dysferlinopathie: Une dystrophie musculaire affectant le muscle strié
2025-09-24 10:00 - 12:00 (America/Edmonton)
1st episode of the RQMO – RARE webinar series. QC presented by Camille Bouchard, PhD student in molecular medicine in the laboratory of Professor Jacques P. Tremblay, Université Laval
Moderator: Jonathan Pratt, Executive Director of the RQMO

Canadian Rare Disease Symposium: Preparing for Clinical Trials – International Regulatory Insights, and cGMP Gene Therapy Manufacturing capabilities in Canada
2025-10-01 13:00 - 16:00 (America/Toronto)
The Stem Cell Network (SCN) and the National Research Council Canada (NRC) are excited to contribute to the national conversation on cell and gene therapy for rare diseases. Building on the 2024 event titled “Canadian Rare Disease Ecosystem Symposium: Genetic Therapies for Rare Disease”, SCN and NRC are pleased to invite you to the Canadian Rare Disease Symposium: Preparing for Clinical Trials – International Regulatory Insights, and cGMP Gene Therapy Manufacturing Capabilities in Canada–a focused half-day event dedicated to advancing gene and cell therapy development in Canada through regulatory alignment and manufacturing readiness.
This virtual symposium will convene international regulatory authorities, Canadian manufacturing experts, and rare disease developers to explore two critical enablers of translational success: clinical trial readiness and cGMP manufacturing capacity.

Rare Diseases International-Community Conversations on Mental Health
2025-10-10 07:00 - 08:00 (America/Edmonton)

A Conversation About Patient Partner Outsider Syndrome
2025-10-15 10:00 - 11:30 (America/Edmonton)
Patient/caregiver partnership in health research has evolved substantially since the launch of Canada’s Strategy for Patient-Oriented Research (SPOR) over a decade ago. However, findings from the Patient Advisors Network (PAN) “Reimagining the Research Landscape” report, commissioned by CIHR in 2024 for the SPOR Refresh, revealed that patient/caregiver partners often feel like outsiders in the research ecosystem. This webinar will take a deeper dive into the OSSU Research Day presentation “Why do patient/caregiver partners feel like outsiders?”
Ms. Maureen Smith will moderate a frank discussion between two of the report authors, Mses. Alies Maybee and Annette McKinnon, and PAN Board member Ms. Sandra Holdsworth, and leading patient-oriented researchers Drs. Manoj Lalu, Ambreen Sayani and Stuart Nicholls, on the barriers uncovered by the report and showcasing best practices to mitigate these barriers.

Aging Out: Transition of care for children with rare neurodevelopmental disorders
2025-10-16 12:30 - 13:30 (America/Toronto)
Approximately one in 12 Canadians are affected by a rare disorder and two thirds of them are children, according to the Canadian Organization for Rare Disorders (CORD). Due to the low number of individual cases, qualified specialists are hard to find. After years of assembling a trusted pediatric care team, parents are often faced with the challenge of finding a new adult care team once their child turns 18. The challenge is even more complex for rare neurodevelopmental disorders requiring multi-disciplinary care.
What strategies would ease the pediatric to adult care transition? This webcast will bring health care providers, medical experts and patient advocates together to share ideas and solutions to bridge the gap between pediatric to adult care for those living with neurodevelopmental disorders.

RareKids-CAN Coffee Chat: Digital Storytelling with Krystle Schofield
2025-10-16 20:00 - 21:00 (America/Toronto)
Join other parents, caregivers, and individuals impacted by rare diagnosis and rare disease on October 16 at 8 P.M. EST. and learn about digital storytelling. Learn from Krystle Schofield, a RareKids-CAN parent partner, B.C. mom, digital storytelling facilitator + photographer.

KTDRR’s 2025 Virtual Knowledge Translation Conference
2025-10-20 13:00 - 2025-10-23 16:00 (America/Toronto)
This conference will look at different ways to make knowledge translation (KT) more effective and impactful. They seek presentations that will discuss how to measure the success of KT efforts, including involving users early on, working with people with disabilities, and customizing products and outreach to meet user needs. They’ll also look for presentations that talk about sharing KT content online, ensuring it’s credible, teaching digital literacy, spotting false information, and making sure disability is represented accurately. In addition, they encourage presentations that explore how technology can boost KT projects, such as using digital tools to build online communities, check text readability, and summarize research. They also welcome presentations that highlight the role of artificial intelligence (AI) in creating easy-to-understand summaries, organizing research, addressing AI bias, and writing effective prompts. Join them to discover new strategies and tools to enhance your KT efforts.

Easing Pain, Advancing Care: A New Pathway for Children with Severe Neurological Impairments
2025-10-29 09:00 - 10:00 (America/Edmonton)
This webinar will provide an overview of the multi-phased project, from clinical trial to implementation. It will highlight the clinical complexity of assessing pain and irritability; the project’s relevance and impact for clinicians, children with health complexity, and their families; as well as key lessons for patient-oriented research and implementation science.

Taking multiple medications? 5 questions to ask
2025-10-29 10:00 - 12:00 (America/Edmonton)
In recognition of Canadian Patient Safety Week 2025 (October 27-31), join Dr. Emily McDonald — a physician specializing in patient safety and Director of the Canadian Medication Appropriateness and Deprescribing Network — for a live, interactive webinar.
Learn essential questions to ask to ensure all your medications are still necessary and helping you.

They are sacred: First Nations' Journey in Autistic Care and Advocacy
2025-10-30 11:00 - 12:00 (America/Edmonton)
UPCOMING RESEARCH FORUM | Grant Bruno, Mariam Ahmad and Tierney Littlechild offer insights on how First Nations families, Elders, autistic individuals, and community leaders are reshaping autism support through Indigenous knowledge, ceremony, and relational approaches.

Screen4Rare: Making AI Diagnosis Understandable
2025-11-04 05:30 - 07:30 (America/Edmonton)
A co-creation workshop inviting non-AI experts to help design communication tools such as videos and brochures that make AI-supported diagnosis more understandable, trustworthy and human.

Canadian Collaborative Conference on Myalgic Encephalomyelitis (ME)
2025-11-04 13:00 - 2025-11-06 17:00 (America/Toronto)
This will include presentations and panel discussions focused on current scientific findings, the opportunities to strengthen research design, and the need to create more inclusive and accessible research spaces.
