Canada is entering a new era in pediatric rare disease research, driven by advances in precision medicine and novel, innovative clinical trial approaches, including gene editing and gene therapies.

RareKids-CAN’s Clinical Trial Navigator network supports pediatric rare disease clinical trial readiness across Canada by helping connect institutions, investigators, and research teams through a coordinated national network.

RareKids-CAN’s Clinical Trial Navigators help strengthen pediatric rare disease clinical trial capacity across Canada by serving as a local contact for investigators, sponsors and clinical trial opportunities.

RareKids-CAN’s Clinical Trial Navigators help strengthen pediatric rare disease clinical trial readiness across Canada by supporting study start-up, facilitating collaboration between institutions, and helping connect investigators and families to research opportunities.

Clinical trials can play a critical role in improving access to treatments and advancing care for children living with rare diseases.

RareKids-CAN, Canada’s pediatric rare disease clinical trials and treatment network funded by the Canadian Institutes for Health Research, supports Bill C-265 which improves access pathways for medical therapies not commercially available in Canada. Introduced by Dr. Marcus Powlowski, Member of Parliament for Thunder Bay – Rainy River, Bill C-265 moves to second reading in Parliament this week.

RareKids-CAN sponsored a dedicated cohort of learners in CanChild McMaster University’s  Family Engagement in Research (FER) Course.

In this Q&A, Busisiwe Zapparoli shares how her background in clinical–developmental psychology and neuropsychology led her to pursue a clinician-scientist path grounded in real-world impact.

In this Q&A, Wallace Wee shares his journey from engineering to medicine and clinical epidemiology, and how that unique path has shaped his approach to pediatric respiratory research.

On February 28th, the RareKids-CAN community came together in a powerful show of collaboration and progress to celebrate Rare Disease Day 2026.