Canadian Rare Disease Network

Webinar: Canadian Survey on the Impact of Disease
2025-05-09 12:00 - 13:00 (America/Toronto)

Webinar for Patient Groups: Evolving Patient Group Input in Drug Reimbursement Reviews
2025-05-08 11:00 - 12:00 (America/Edmonton)

Save the date: Redefining Patient Engagement
2025-05-08 08:00 - 17:00 (America/Denver)

TruePKU Meeting
2025-05-08 08:00 - 17:00 UTC
Mark your calendar for May 8, 2025, for the TruePKU meeting – a first-of-its-kind opportunity for members of the PKU community to share their experiences, needs, and treatment priorities directly with the U.S. Food and Drug Administration, life science companies with active PKU programs, researchers, and healthcare professionals.

2025 NMD4C Basic Research Summer School
2025-05-07 08:00 - 2025-05-08 17:00 (America/New_York)
The annual NMD4C summer school provides lecture-based and hands-on training workshops to neuromuscular trainees in cutting-edge wet lab techniques, while providing an environment for trainees to present their research and network in-person with colleagues and senior investigators from across Canada.

Medicines Reimagined: Unlocking the Potential of Existing Drugs for Patient Benefit
2025-05-07 08:00 - 2025-05-08 17:00 (Europe/Helsinki)
Join iDR25 – the international drug repurposing conference – to unlock the full potential of medicines by driving innovation for patient benefit. From cutting-edge AI approaches transforming drug discovery to exploring innovative business models and regulatory pathways for market access

Palliative Care in Chorea and Huntington's Disease
2025-05-06 19:00 - 20:00 (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).

Applying Principles of Equity, Diversity, Inclusion, and Accessibility in Patient Engagement with Equity Seeking Groups
2025-05-05 15:00 - 17:00 (America/New_York)

FSHD 360 Conference
2025-05-03 08:00 - 17:00 (America/Toronto)
FSHD research and drug development is in a new era. Do you know what’s happening and how this might impact your life? Here’s your chance to hear from the research and advocacy leaders in Ontario as well as from biopharmaceutical companies that are working on FSHD therapies. Get answers to your questions about your health, maintaining optimal function through physical therapy, exercise, assistive devices, and more. Don’t miss out on the opportunity to meet patients and families and network with FSHD Society leaders and activists in the community!

FSHS 360 Conference
2025-05-03 08:00 - 17:00 (America/Toronto)
Patients and family members affected by FSHD are invited to attend an event hosted by the FSHD Canada Foundation and the FSHD Society! The FSHD 360 Conference Ontario is an event where patients and family can meet leaders in FSHD research and hear the latest updates, including presentations from NMD4C investigators Dr Lawrence Korngut and Dr Hanns Lochmuller.

Managing Anxiety in Children and Adolescents with Neurodevelopmental Disorders (NDDs)
2025-05-01 18:30 - 20:30 (America/Edmonton)
Presenters: Thomas Qiao, MSc, Research Coordinator, Department of Psychiatry, University of Calgary Noah Furlani, MSc, Research Coordinator, Department of Psychiatry, University of Calgary Dr. Kara Murias, MD PhD FRCPC, Pediatric Neurologist, Alberta Children’s Hospital; Assistant Professor, Department of Pediatrics and Clinical Neuroscience, University of Calgary

Myotonic Dystrophy Foundation Conference 2025
2025-05-01 08:00 - 2025-05-04 17:00 (America/New_York)
MDF invites you to attend the Professional Track at the 2025 MDF Conference in Indianapolis, Indiana from May 1st to May 4th! Sometimes called the “DM family reunion” – the MDF Conference is an event to unite the DM community in our journey towards treatments and a cure for myotonic dystrophy (DM). This year’s program includes educational sessions exploring research, drug development, symptom management, and other critical topics for DM families, plus opportunities for formal and informal networking, wellness, and fun!

Genome BC’s Genomics Forum 2025
2025-04-30 08:00 - 17:00 (America/Vancouver)

Neurodiversity in the Workplace: 6 Principles for Inclusion
2025-04-29 10:00 - 11:00 (America/Edmonton)
Learn how to create a more inclusive workplace for neurodivergent employees with our 6 principles!

Save the Date: Rare Disease Day 2025 Conference
2025-04-29 08:00 - 2025-05-01 17:00 (America/Denver)

Health Data for All of Us: A Public Dialogue on AI in Health
2025-04-29 08:00 - 16:00 (America/New_York)
Join HRDN conversation about key ethical, equity and privacy challenges related to AI and health!

Health Data for All of Us: A Public Dialogue on AI in Health
2025-04-29 08:00 - 16:00 (Asia/Karachi)
Join the conversation about key ethical, equity and privacy challenges related to AI and health!

Canadian Rare Disease Innovation Showcase 2025
2025-04-28 16:00 - 20:15 (America/Chicago)
Join us on Monday, April 28, 2025, at The Westin Hotel in downtown Calgary (or virtually) for an inspiring afternoon dedicated to innovation, collaboration, and progress in rare disease research and care.
This event will spotlight innovative advancements and inspiring stories from Canada’s rare disease community. Through engaging lightning talks and networking opportunities, you’ll connect with experts, exchange ideas, and be part of the movement driving meaningful change.
Every discovery has the power to change lives. Be part of the conversation and help shape the future of rare disease care in Canada.

Summit 2025: Ending The Diagnostic Odyssey
2025-04-28 08:00 - 2025-04-30 17:00 UTC | washington, WA, United States
A first-of-its kind collaborative event for Undiagnosed, Ultra-Rare & Rare disease industry, clinical and community leaders.
The diagnostic odyssey for rare diseases is a significant challenge, often leaving patients and families lost in a maze of tests, misdiagnoses, missed opportunities to participate in clinical trials, and delayed treatment. Summit 2025 brings together a diverse group of stakeholders – patient advocates, industry leaders, policymakers, clinicians, researchers and payers – to chart a collaborative path towards a future where timely and accurate diagnoses are a reality for all with rare diseases.

Mitochondrial Transplantation and Next Generation Therapeutics Conference
2025-04-27 08:00 - 2025-04-29 17:00 (America/Halifax)
Join Northwell Health on April 27-29, 2025, at Hofstra University on Long Island (NY) for a unique forum for the presentation and discussion of new research developments in mitochondrial transfer and transplantation.

Navigating Conflict with Care: Building Stronger Patient Engagement Teams in Research
2025-04-25 11:30 - 12:30 (America/Toronto)
​Conflict is a natural part of teamwork—but in family engagement in research, how we navigate it matters. Join them for a compassionate conversation on resolving conflicts in family engagement teams, fostering trust, and strengthening collaboration.

Lunch N’ Learn: Talking Research with Patient Partners
2025-04-24 12:00 - 13:30 (America/Toronto)
Join this virtual workshop on Zoom on April 24, 2025, at 12:00 p.m. (EST) to develop communication skills for engaging with patient partners in research.
Hosted by the HBHL EDI Trainee Committee, the session features medical librarians and a patient advocate sharing insights on plain language and patient-oriented research.
Presenters:

Sabrina Burr, MISt – Librarian of the Neuro-Patient Resource Centre at The Neuro-MNI
Paule Kelly-Rhéaume – Medical librarian at the McGill University Health Centre
JoAnne Mosel, CPA – Advocate in the Rare Disease space with over 30 years of experience

Limb-Girdle Muscular Dystrophy Masterclass
2025-04-24 08:00 - 2025-04-25 17:00 (America/New_York)
Are you involved in diagnosing or managing Limb-Girdle muscular dystrophy (LGMD)? This April 2025, join TREAT-NMD™ in Montreal, Canada, for an in-person educational event designed for healthcare professionals in the neuromuscular field.

Neuropsychiatry & Mental Health 2025
2025-04-24 08:00 - 2025-04-25 17:00 UTC
Theme: Innovations in Neuropsychiatry and Evolution in Medicines of Neurological Disorders for Better Mental Health.

ACE Inhibitors as a Cardioprotective Treatment in Dystrophinopathies.
2025-04-17 10:00 - 11:00 (America/Halifax)
Join C-PATH on April 17, noon ET, for an insightful presentation by Dr. Karim Wahbi, Professor of Cardiology at the University of Paris and head of the Reference Center for Neuromuscular Cardiomyopathies. Dr. Wahbi specializes in the clinical management of cardiomyopathies, with a focus on neuromuscular diseases like Duchenne muscular dystrophy.

Thinking Outside of Your In Box: Innovative Fundraising Opportunities
2025-04-10 14:30 - 16:00 (America/New_York)
Leadership in rare disease advocacy requires creativity and resourcefulness. This session highlights unconventional fundraising strategies and case studies of successful campaigns. Participants will discover how to secure funding through innovative approaches, such as partnering with non-traditional sponsors, and receive actionable tips to implement these ideas in their own organizations.

RDI: Leveraging the EML for Access to Rare Disease Treatments
2025-04-10 14:00 - 15:00 UTC
The WHO Essential Medicines List (EML) is a critical instrument for advancing Universal Health Coverage (UHC) by ensuring equitable access to essential medicines. However, its role in rare disease treatment remains under-recognized. This webinar will explore how the EML can serve as a powerful tool to improve access to treatments for persons living with a rare disease (PLWRD) at both global and national levels.
Webinar Objectives:

Gain a deeper understanding of the WHO Essential Medicines List and its significance for rare diseases
Learn about the findings from RDI’s Essential Medicine Working Group on challenges, best practices, and opportunities for regarding the application process of EML
Hear about call-to-action to enhance global and national access to essential medicines

RareKids-CAN Annual Conference
2025-04-10 08:00 - 2025-04-11 17:00 (America/New_York)
The mission of the RareKids-CAN: Pediatric Rare Disease Clinical Trials and Treatment Network Strategic Planning Conference is to collaboratively shape the future of pediatric rare disease research and clinical trial capacity in Canada. By bringing together over 100 patient and family partners, researchers, sub-platform leaders, and key stakeholders, they aim to co-develop a shared vision that accelerates pediatric drug development and progress in clinical trials, improves treatment access, and strengthens national and international partnerships.

Five Good Ideas to support and benefit from working caregivers in your workplace
2025-04-09 11:00 - 12:00 (America/New_York)

13th ISSAID Meeting
2025-04-08 08:00 - 2025-04-10 17:00 (Europe/Helsinki) | paris, Paris, France
13th Meeting of the International Society of Systemic Autoinflammatory Diseases (ISSAID), which will take place in Paris from April 8th to 10th, 2025, at the “Cité Internationale” – a magnificent haven of peace near many transport amenities where you will particularly enjoy the sumptuous Honorat lounge overlooking a lush green park.
April in Paris offers a delightful mix of mild weather, budding greenery, and a relaxed atmosphere, perfect for strolling along the Seine after a day of work.
The ISSAID 2025 meeting will provide an opportunity for pediatric and adult rheumatologists, immunologists, general pediatricians and internists, scientists, and others interested in systemic autoinflammatory diseases to come together to share new findings related to autoinflammation and to learn about recent advances in this field.
The ISSAID 2025 meeting will highlight the topic of neurologic inflammation, and we hope it will facilitate better assessment, care, and research on this particularly complex subject. We will present current topics such as artificial intelligence, mechanisms of inflammation, and new diseases.
The ISSAID 2025 meeting will promote interactions between old and new generations so that the science of SAIDs can be transmitted and best adapted to societal transformations and the challenges of tomorrow’s medicine.
