This past Rare Disease Day on February 28, 2025, our team came together to raise awareness, share patient and family experiences, and celebrate the work we’ve done so far while acknowledging the work yet to come as a network.

Congratulations to network members Maureen Smith, Dr. Beth Potter and Dr. Lauren Kelly for receiving IHDCYH competition awards!

MICYRN (and by association RKC) officially signed an MoU with ECRIN, bringing us new opportunities for international collaborations.

RareKids-CAN is thrilled to congratulate Maureen Smith on her appointment to the Board of Directors for Canada’s Drug Agency (CDA) as its first-ever Patient Member!

A dedicated patient partner with RareKids-CAN, Maureen brings extensive leadership experience, including her role as co-Principal Investigator and Patient Engagement Strategy lead with INFORM RARE.

Andrea and her son Jakob, one of our incredible family partners, recently traveled to the U.S. to participate in a rare disease clinical trial, accessing a treatment not yet available in Canada. Their inspiring story highlights the urgent need for better access to clinical trials and treatments within Canada.

RareKids-CAN remains committed to transforming the Canadian rare disease landscape, ensuring more patients can receive lifesaving treatments at home.

On November 25-26, the Health Canada Relations and Advocacy Sub-Platform of RareKids-CAN and MICYRN, in partnership with the SickKids Child Health Policy Accelerator, gathered researchers in Ottawa to advocate for regulatory changes in pediatric clinical trials. Meetings with Health Canada leaders and over 250 staff emphasized the need for a regulatory exemption for off-label use of marketed Standard of Care therapies in pediatric trials. The proposal was well received, and engagement with the federal government will continue.

RareKids-CAN and the Maternal Infant Child and Youth Research Network (MICYRN) are proud to support the launch of the European Rare Disease Research Alliance (ERDERA) as a formal Associate Partner. In partnership with the Canadian Rare Disease Network (CRDN), RareKids-CAN co-leads the Canadian National Mirror Group (NMG), aligning Canada’s rare disease initiatives with global strategies. This collaboration will enhance international partnerships, advance research coordination, and improve outcomes for individuals with rare diseases.