Sara Pot reflects on her experience, key learnings, and the opportunities she sees for strengthening patient and family engagement in Canada's rare disease landscape. 

RareKids-CAN was pleased to sponsor two of our Clinical Trial Navigators, Cara Grobbecker and Faiza Khawaja to attend the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting virtually.  

The Canadian National Mirror Group, led by RareKids-CAN, is helping ensure Canadian perspectives are represented in international efforts to strengthen rare disease research and data infrastructure.

Cell and gene therapies are rapidly transforming the pediatric rare disease treatment landscape and are offering new possibilities for conditions that have historically had few or no effective treatment options. 

RareKids-CAN Nominated Principal Investigator and MICYRN Scientific Director Dr. Thierry Lacaze recently joined international leaders in a rare disease research, regulation, and drug development in Boston

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.