Canadian Rare Disease Network

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.

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.

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.

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.

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.

17th International Symposium on Sjögren's Disease
2026-09-29 08:00 - 2026-10-02 17:00 (Europe/Paris)
This prestigious event, which brings together researchers, physicians, patients and experts from around the world to discuss the latest advancements in Sjögren’s disease.
