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.

4th Annual Scientific Symposium on SPG4
2026-03-25 08:00 - 2026-03-27 17:00 (America/Toronto)
The premier gathering for childhood-onset SPG4 research returns in 2026 — uniting leading scientists, clinicians, industry innovators, patient advocates, and families for three days of focused collaboration. Designed to accelerate the path from discovery to treatment, the symposium will feature experts from across the U.S. and abroad, offering a rare opportunity to share knowledge, align priorities, and drive progress.

CPD-Accredited Webinar | Duchenne Muscular Dystrophy in the New Steroid Age: Practical Considerations
2026-04-07 15:00 - 17:00 (America/Edmonton)
NMD4C, MDC, and Defeat Duchenne Canada are pleased to invite you to a CPD-accredited webinar on Duchenne Muscular Dystrophy in the Evolving Steroid Era: Practical Neuromuscular and Endocrine Considerations. This CPD-accredited webinar is primarily targeted at Canadian clinicians, academics and trainees with an interest in neuromuscular diseases.

MitoRevolution Research Symposium and Innovation Day
2026-04-09 08:00 - 17:00 (America/Toronto)
Researchers, clinicians, and trainees working in mitochondrial science are invited to attend the 6th Annual MitoRevolution Research Symposium and Innovation Day, hosted by their partners Mito2i, on Thursday, April 9, 2026, at the MaRS Auditorium in Toronto.

Ehlers-Danlos Syndrome (EDS) Research Symposium
2026-04-09 08:00 - 2026-04-10 17:00 (America/Edmonton)
The Ehlers-Danlos Syndrome (EDS) Research Symposium is a two-day event, April 9-10, designed to bring together clinicians, researchers, students, patients, and community partners to examine current research, clinical challenges, and emerging approaches to the diagnosis and management of the Ehlers-Danlos syndromes.⁠
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ALS Clinical Trials Unboxed
2026-04-10 13:00 - 14:00 (America/Toronto)
On Friday, April 10, at 1:00 p.m. ET, tune into ALS Clinical Trials Unboxed to learn about ongoing ALS clinical trials in Canada and globally for people living with ALS. In this webinar, Dr. Tyrell J. Simkins, DO, PhD, Senior Director of Clinical Development at Coya Therapeutics, will discuss the scientific rationale behind COYA 302 and the design of the ALSTARS trial.

CORD: Rare Disease Day 2026 Conference
2026-04-29 08:00 - 2026-04-30 17:00 (America/Toronto)
Join CORD for their Annual Rare Disease Day Conference in Toronto!

Ehlers-Danlos Society: Biobank Research Event
2026-05-04 08:00 - 2026-05-08 17:00 (America/Los_Angeles)
This initiative supports research into the different types of Ehlers-Danlos syndromes (EDS) and hypermobility spectrum disorders (HSD) by collecting biological samples and health information from people with and without these conditions. ⁠

International Drug Repurposing Conference 2026
2026-05-12 08:00 - 2026-05-13 17:00 (Europe/Brussels)
Join the leading international drug repurposing conference to explore the future of patient-centric medicines repurposing.
From rare diseases and AI-driven drug discovery, to policy, funding and community-led success stories, iDR26 brings together the global drug repurposing community to navigate the next chapter in maximising patient benefit.

ALS Canada: ALS Clinical Trials Unboxed
2026-05-15 10:00 - 11:00 (America/Edmonton)
On Friday, May 15, 2026 at 12 p.m. ET, join ALS Canada for our next ALS Clinical Trials Unboxed webinar to learn more about ongoing ALS clinical trials.

RQMO: Le grand pouvoir des petites populations
2026-05-19 10:00 - 11:00 (America/Edmonton)
6e épisode de la série de webinaires RARE.QC-RQMO présenté par Simon Girard, Ph.D, Professeur en génétique humaine, Université du Québec à Chicoutimi et un de ses étudiants au doctorat en biologie, M. Gilles-Philippe Morin.
Le titre de la présentation de M. Morin est : Déceler la structure fine de population du Saguenay–Lac-Saint-Jean par la génétique et la généalogie.
Modération : Jonathan Pratt, Directeur général du RQMO

Rare Diseases International: A Catalyst for Global Health Transformation
2026-05-20 10:00 - 11:30 (America/Edmonton)
Bringing together policymakers, patient advocates, industry leaders, researchers, and global health institutions, the event will explore how rare diseases can drive people-centered, equitable health systems.

Sickle Cell Awareness Group of Ontario: Learning for Life Webinar
2026-05-30 09:00 - 22:30 (America/Edmonton)
Join SCAGO for an informative virtual session on Novel Therapies in Sickle Cell Disease Pain Management: What Patients and Families Should Know.

ECRD: Rare Diseases in a Changing & Competitive Europe: Shaping policies to address the unmet needs of people living with rare diseases
2026-06-02 08:00 - 2026-06-04 17:00 (Europe/Prague)
The ECRD is the largest, patient-led, rare disease policy-shaping event held in Europe.
By bringing together people with rare diseases and patient advocates, policy makers, healthcare industry representatives, clinicians, regulators and Member State representatives, EURORDIS harnesses the power of this extensive network to shape goal-driven rare disease policies of the future.

Planning Orphan Device Development Using the GAITS Framework
2026-06-02 09:00 - 10:00 (America/Toronto)
This webinar kicks off a new joint EU4Health webinar series on paediatric and orphan devices! Three EU4Health projects, i4Kids4Rare, DeCODe, and OrphaDev4KIDS have joined forces to deliver a series of expert webinars on planning, regulation, and clinical pathways for paediatric and orphan devices.

RareKids-CAN Advanced Therapy Medicinal Products (ATMPs) series: ATMP Webinar: A Practical Primer for Clinical Coordinators, Nurses & Pharmacists
2026-06-15 13:00 - 14:00 (America/Edmonton)
Designed for clinical coordinators, nurses, pharmacists, and research staff working in pediatric rare disease trials, this introductory session will provide a practical overview of:
🔹 Gene therapies, cell therapies, and antisense oligonucleotides (ASOs)
🔹 How ATMPs differ from traditional therapies
🔹 Key operational considerations for clinical teams
🔹 Strategies for explaining ATMPs in clear, accessible language

ERDERA Clinical Trial Call 2026: Information Webinar
2026-07-06 07:00 - 09:00 (America/Edmonton)
ERDERA will host an informational webinar on 6 July 2026. During this session, participants will have the opportunity to hear directly from ERDERA experts, who will present the objectives, scope, and requirements of the call, and address questions from prospective applicants.

CIHR: Information Webinar for Canadian applicants to the ERDERA Clinical Trial Call 2026
2026-07-07 10:00 - 11:00 (America/Toronto)
CIHR is hosting an information session to provide interested Canadian applicants with information on the requirements of the “ERDERA Clinical Trial Call 2026 (ECTC 2026)” funding opportunity and to answer questions on how to apply.

Dysautonomia International: Piece by Piece: Building the Future of Dysautonomia Care
2026-07-09 08:00 - 2026-07-12 17:00 (America/Chicago)
Join Dysautonomia International in Houston, Texas, July 9–12, 2026 for the 14th Annual Dysautonomia International Conference, the world’s largest conference on autonomic disorders.

IMPaCT Webinar: Responsible Research Practices: How Reporting Guidelines Can Improve Child Health Trials
2026-07-09 12:00 - 13:00 (America/Toronto)
Register today for IMPaCT’s next webinar with Ami Baba, PhD, where she’ll discuss Responsible Research Practices: How Reporting Guidelines Can Improve Child Health Trials.
✅ Identify who needs trial reporting guidelines
✅ Introduce pediatric-specific reporting guideline extensions for trials and systematic reviews
✅ Describe how SPIRIT-C, CONSORT-C, TIDieR-C, and PRISMA-C can impact both upstream and downstream research processes

RareKids-CAN: Delivering Gene Therapy Trials: Understanding Clinical Workflows, Operations, and the Patient Journey
2026-07-22 12:00 - 13:00 (America/Toronto)
Join RareKid-CAN on July 22nd for their second webinar in our ATMP series for research coordinators, nurses, pharmacists, investigators, and other clinical research professionals.
Their speaker Ana Stosic will provide a practical overview of the clinical workflows involved in delivering gene therapy clinical trials for pediatric rare diseases.

Health Canada Webinar: Real-World Evidence in the National Strategy for Drugs for Rare Diseases
2026-07-22 12:00 - 13:00 (America/Toronto)
This session is designed to provide you with a clear understanding of the goals, an update on progress, and the next steps as planning advances. It will include a short presentation from Health Canada followed by a Question & Answer session.

Pulmonary Hypertension Association Canada’s PH Community Conference & Scientific Sessions
2026-09-24 08:00 - 2026-09-26 17:00 (America/Vancouver)
🔬 Thursday, September 24
CPHPN and CaPH-Net meetings (by invitation) and the Scientific Sessions Opening Reception. Primarily for healthcare professionals, researchers, and invited network members.
🩺 Friday, September 25
Scientific Sessions — a full day dedicated to the latest PH research, clinical advancements, and collaboration among healthcare professionals and researchers. The day concludes with the Patient & Family Symposium Welcome Reception, where the entire PH community comes together.
💜 Saturday, September 26
Patient & Family Symposium — designed specifically for PH patients, caregivers, family members, and supporters. A day of education, inspiration, connection, and community.
