Ottawa NMD 2025 Conference
2025-09-11 08:00 - 2025-09-13 17:00 (America/Toronto)
7th Ottawa International Conference on Neuromuscular Disease and Biology will take place at the Fairmont Château Laurier in Ottawa, Canada on September 11-13, 2025.
The Ottawa NMD 2025 conference will offer an outstanding program emphasizing recent breakthroughs in basic and translational research and clinical discoveries in neuromuscular disease.

Undiagnosed Hackathon 2025
2025-09-21 08:00 - 2025-09-24 17:00 (America/Chicago)
350 million people live with an undiagnosed disease worldwide. Genome sequencing can provide answers for 40% of the People Living With Undiagnosed Disease (PLWUD) but around 60% still don’t receive a diagnosis. The purpose of the Undiagnosed Hackathon is trying to find new ways and collaborations to solve the undiagnosed diseases, that can’t be solved today. In this unique event multidisciplinary teams of clinicians, bioinformaticians/molecular biologists, scientists, developers, AI specialists and brilliant minds will work together to tackle the most difficult diseases.

6th Western Canadian Neuromuscular Conference
2025-09-25 08:00 - 2025-09-27 17:00 (America/Edmonton)
The 6th Western Canadian Neuromuscular
Conference is confirmed for 2026! Don’t miss
the opportunity to network with leading experts
and stay at the forefront of medical
advancements in the field of neuromuscular
diseases.

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.

CAGC & CCMG Joint Conference
2025-10-05 08:00 - 2025-10-08 17:00 (America/Edmonton)
The CAGC & CCMG Joint Conference will be a unique gathering of medical and laboratory geneticists and genetic counsellors with diverse expertise, all sharing a common interest in education and advocating for best practices in clinical and laboratory genetics. Through presentations, workshops, breakout sessions, abstract sessions, and engagement with industry partners, the event propels forward clinical practice, scientific advancements, and excellence across all aspects of genetics in Canada.

30th Annual International Congress of the World Muscle Society
2025-10-07 08:00 - 2025-10-11 17:00 (Europe/Vienna)
The World Muscle Society is a dynamic community that aims to promote, disseminate, and share all aspects of neuromuscular physiology and diseases, from basic science to patient care. It encompasses a broad range of scientists and healthcare professionals who share the common interest for neuromuscular disorders.

The World Orphan Drug Congress
2025-10-27 08:00 - 2025-10-29 17:00 (Europe/Amsterdam)
The World Orphan Drug Congress is the largest and most established orphan drug & rare disease meeting of its kind across the globe.
From cell and gene therapy, genetic testing, and market access, to real world evidence, this one meeting covers the whole orphan drugs value chain where science, government and manufacturers all come together to create groundbreaking progress.

Cardiac Precision Medicine in the 21st Century
2025-10-29 08:00 - 2025-10-31 17:00 (America/Toronto)
The 10th International Cardiovascular Genomic Medicine & SickKids Cardiac Precision Medicine Conference is making its debut in North America! This year’s theme, “Cardiac Precision Medicine in the 21st Century,” delves into cardiovascular genomics and precision medicine in the era of ‘Omics’, AI and digital medicine. Join us as we bring together international experts and innovators to explore transformative research and collaborative opportunities that can shape the future of cardiovascular medicine.
This event will feature the 4th William Harvey Oration delivered by Dr. Sharlene Day and the Dr. Michael Braudo Lectureship delivered by Dr. Ma’n H. Zawati.

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.

ITINERARE Symposium 2025
2025-11-18 08:00 - 17:00 (Europe/Zurich)
The programme features experts from a variety of fields, ranging from basic and translational research to clinical application and the ethical, social and legal aspects of rare disease research.

2025 IMPaCT Summit
2025-11-18 08:00 - 2025-11-19 17:00 (America/New_York)

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.

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.

Webinar: Lymphodepletion for CAR-T/IEC Outcomes
2025-12-17 12:00 - 14:00 (America/Toronto)
Achieving consistent and effective outcomes in CAR T and other immune effector cell (IEC) therapies relies heavily on getting lymphodepletion “right.” Emerging evidence shows that both under- and over-exposure can impact safety, efficacy, and long-term persistence, making optimized dosing strategies increasingly critical as the field evolves.
This webinar will explore how model-informed and data-driven approaches can help identify the optimal lymphodepletion exposure window, improve predictability, and support more standardized practices across patient populations and treatment centers. Speakers will discuss current challenges, scientific rationale, clinical insights, and practical considerations for implementation.

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.

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.

CATALIS-Clinical Research 101: Webinar 1
2026-01-22 18:00 - 18:30 (America/Toronto)
Webinar 1 on January 22, 2026 from 6 pm to 6:30 pm

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.

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

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.

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.

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.

Canada's Drug Agency: How Global Policy Changes Are Reshaping HTA for Rare Diseases
2026-02-19 13:00 - 14:30 (America/Edmonton)
HTAi’s Rare Disease Interest Group will celebrate 2026 Rare Disease Day with a conversation on how global policy changes are reshaping HTA processes for rare disease technologies. The webinar aims to update the global HTA community on recent advances in rare disease policy, particularly following the adoption of the World Health Assembly (WHA) Resolution on Rare Diseases, and to explore its implications for HTA systems globally. The multi-stakeholder expert panel will discuss how global policy commitments can translate into meaningful regional, national, and local action within HTA. This event is the inaugural webinar in a year-long webinar series, catalyzing discussions on how legislative changes in health systems are reshaping HTA for rare diseases through regional real-world case studies and examples.

Stem Cell Network: Clinical Translation in Cell and Gene Therapy
2026-03-04 11:00 - 12:30 (America/Edmonton)
Stem Cell Network and BioCanRx are pleased to present this webinar on clinical translation, offering trainees, early-career investigators, and researchers a high-level introduction to how cell and gene therapies (including regenerative medicine and cancer biotherapeutics) move from the lab to clinical trials.

RareKids-CAN: Rare Methods Workshop Series: Eliciting prior distributions for rare disease clinical trials
2026-03-11 13:00 - 14:00 (America/Toronto)
Are you a rare disease methodologist, investigator or trainee? Join RareKids-CAN and speaker Arlene Jiang for a Rare Methods Workshop on expert elicitation in rare disease research on March 11.
Learn what expert elicitation is, how it can support rare disease clinical design and how expert input can be used to construct a prior distribution.

Rare Disease Summit: Access USA
2026-03-17 08:00 - 2026-03-19 17:00 (America/Edmonton)
The Rare Disease Summit unites stakeholders to accelerate therapeutic innovation for areas of unmet medical need. Gain actionable insights on launch planning, market access, reimbursement strategies and strategic partnerships to optimize rare disease commercialization and elevate patient impact.

MAINFRAME Symposium on AI-Driven Small-Molecule Drug Discovery
2026-03-18 08:00 - 2026-03-19 17:00 (Europe/Rome)
Co-organized by Conscience in partnership with The Structural Genomics Consortium (SGC) and local organizers at the Vall d’Hebron Institute of Oncology (VHIO), the Barcelona Supercomputing Center (BSC), and the Institute of Research in Biomedicine (IRB), this two-day in-person symposium will bring together the global community working at the intersection of artificial intelligence, computational chemistry, and small-molecule drug discovery.
