IMPaCT RareKids-CAN logos Text reads Congratulations to Busisiwe Zapparoli and Wallace Wee, IMPaCT-RareKids-CAN trainess! Photo of Wallace Wee an Asian man with glasses and a black shirt with red and white striped tie, Photo of Busisiwee Zapparoli a Black woman with long braids and a stripped blouse

Congratulations to Busisiwe Zapparoli and Wallace Wee, IMPaCT–RareKids-CAN trainees!

Read how Busisiwe Zapparoli is improving outcomes for patients: www.rarekidscan.com/news/impact-...

Read how Wallace Wee is advancing pediatric lung disease research: www.rarekidscan.com/news/impact-...

"I look forward to gaining valuable insights and sharing key takeaways with RareKids-CAN to contribute to national ATMP trial readiness efforts" - Cara Grobbecker, Clinical Trial Navigator, RareKids-CAN, Research Coodrinator, Children's Hospital London Health Science Centre

"I'm grateful for the opportunity to attend the ASGCT Annual Meeting and engage with inspiring work in pediatric gene therapy." - Faiza Kawaja, Clinical Trial Navigator, RareKids-CAN; KINDTrials Netowrk Manager, Research manager autism research centre, Hollan Bloorview Kids Rehabilitation Hospital

Congratulations to Cara Grobbecker and Faiza Khawaja, the awardees of RareKids-CAN’s sponsored opportunity to attend the @asgct.bsky.social Annual Meeting.

#RareDisease #ClinicalTrials #Pediatrics #HealthInnovation

World Health Day 2026 text over an illustration of two doctors and one clinician.

It's World Health Day and RareKids-CAN is proud to stand behind this year’s theme: Together for health. Stand with science. Today, we celebrate the power of collaboration and recommit to building a future where every child benefits from the best that science and healthcare can offer. #WorldHealthDay

RareKids-CAN Nominated Principal Investigator Thierry Lacaze, delivered the keynote address at the @rare-qc.bsky.social Scientific Day.

He underscored our initiatives and highlighted the critical role of advancing research + collaboration in the pediatric rare disease space.

#PediatricRareDisease

Access Pathways for Therapies Unfit for Commercialization Advance initiatives that support the development and implementation of therapies that are unlikely to be commercially viable but offer meaningful benefits for rare disease patients and their families.

Global Engagement Continue to build on international collaborations to increase access to innovative PRDCTs and improve pediatric rare disease outcomes in Canada.

Regulatory Pathways Identify policy objectives that promote a competitive Canadian regulatory environment aligned with international best practices and reduce barriers to pediatric rare disease treatments.

RareKids-CAN is committed to enabling timely access to pediatric rare disease therapies in Canada. Our third strategic priority is all about driving regulatory reform and system innovation.

Read more here
static1.squarespace.com/static/662c1...

#RareDiseaseResearch #PediatricRareDiseases

Why Caregiver Voices Matter in Global Rare Disease Research  — RareKids-CAN For families affected by rare diseases, the path to answers is seldom straightforward. Diagnosis can take years, treatments are often limited, and navigating healthcare systems can be overwhelming.&nb...

Genevieve Currie and Leah Sarah Peer have been selected to join the James Lind Alliance Steering Committee. They will bring lived experience directly into conversations that define future research priorities

Read more:
www.rarekidscan.com/news/why-car...

#RareDisease #PediatricRareDisease

Last month, the RareKids-CAN community came together to celebrate Rare Disease Day 2026 with events across the country

Read more about the impact and activities here:
www.rarekidscan.com/news/rare-di...

#RareDiseaseDay #PediatricRareDisease

National Expertise Catalogue: Develop and maintain a centralized database of methodological and RD clinical experts to assist with protocol development and site and investigator identification.

Registry Development Support: Advance the development, maintenance, and use of patient registries and real-world data to support the full pediatric rare disease clinical trials life cycle.

Trial Matching & Patient Identification Develop a centralized, data-driven portal that supports trial planning, attracts new PRDCTs, and enables optimized patient-trial matching across Canada to enhance PRDCT visibility and feasibility.

RareKids-CAN's second strategic priority aims to optimize participant, site, and trial matching.

Read more here: static1.squarespace.com/static/662c1...

#RareDiseaseResearch #PediatricRareDiseases #ClinicalTrials

Strengthening Global Bridges: Canada’s Leadership in Rare Disease Research at the ERDERA Sofia Meeting — RareKids-CAN The Canadian National Mirror Group continues to play an important role in connecting Canada’s rare disease ecosystem with global initiatives

RareKids-CAN participated in the ERDERA meeting in Sofia 🇨🇦🌍

Presentations highlighted how Canada connects research, policy, and patient voices -- while engaging global partners in pediatric rare disease research and care.

Read more: www.rarekidscan.com/news/strengt...

#RareDisease #GlobalHealth

RareKids-CAN leaders Breanne Stewart & Dr. Thierry Lacaze shared a vision for advancing pediatric clinical trials at the ACCESS Annual Meeting in Toronto.

Read more: www.rarekidscan.com/news/rarekid...

#PediatricResearch #ClinicalTrials #RareDisease #ATMP

Following the release of our Strategic Plan, RareKids-CAN is sharing our first strategic priority: Strengthening PRDCTs Capacity and ATMPs Readiness.

Read more here: static1.squarespace.com/static/662c1...

#RareDiseaseResearch #PediatricRareDiseases #ClinicalTrials

New legislation introduced in Parliament today aims to improve access to essential medications not available in Canada.

Learn more about the tabled bill here: www.rarekidscan.com/news/rarekid...

#HealthPolicy #AccessToMedicines #RareDisease #CDNPoli

We’re pleased to share the revised 2025-2029 Strategic Plan!

We’re grateful to our patient and family partners, researchers, institutions, and collaborators who shaped this evolution.

Read the full plan here: static1.squarespace.com/static/662c1...

#RareDiseaseResearch #PediatricRareDiseases

This International Women’s Day, we celebrate the women driving change in pediatric rare disease research.

At RareKids-CAN, we’re proud to work alongside extraordinary women across Canada who are accelerating the rare disease space.

#InternationalWomensDay #IWD2026 #PediatricRareDisease

If you're a rare disease methodologist, investigator, or trainee, join us on Mar 11 at 1pm EST for a Rare Methods Workshop on Expert Elicitation in Rare Disease Research.

We hope to see you there! Register today: us02web.zoom.us/meeting/regi...

#RareDisease #ClinicalResearch #ResearchWorkshop

Pediatric rare diseases may affect small numbers at a time, but together they represent a major challenge.

With RareKids-CAN, McMaster researchers & family leaders are building a more coordinated national approach to rare disease trials.

pediatrics.healthsci.mcmaster.ca/mcmaster-exp...

Institutions across Canada are raising awareness of Rare Disease Day on February 28th. BC Children’s Hospital, the University of Alberta, and the Canadian Rare Disease Network are all hosting events. To register or learn more visit www.rarekidscan.com/events

#RareDiseaseDay #PediatricRareDisease

Rare Disease Day 2026 : Raising awarness for the 200 million children impacted by a rare disease

Feb 28 is Rare Disease Day.

400M people worldwide are impacted by rare diseases, 200M are children. RareKids-CAN is accelerating access to pediatric rare disease trials and treatments across Canada. Find out how at www.rarekidscan.com

#RareDiseaseDay #HealthEquity #PediatricRareDisease

RareKids-CAN Rare Methods Workshop Series: Eliciting Prior Distributions for Rare Disease Clinical Trials March 11 | 1pm EST | Virtual | Speaker: Arlene Jiang, Member of the RareKids-CAN Statistical Methods Sub-Platform, Biostatistical Analyst, EMBaRC Lab, SickKids

Are you a rare disease methodologist, investigator or trainee? Join us for a Rare Methods Workshop on expert elicitation in rare disease research on March 11 at 1pm EST.

Register today: us02web.zoom.us/meeting/regi...

#RareDisease #ClinicalResearch #ResearchMethods

Did you miss our most recent webinar?

Watch the recording of From Evidence to Access: Advancing Pediatric Rare Disease Drug Availability in Canada with Matthew McDonald, Douglas Clark, Maryam Oskoui, and Marc-André Gagnon here: www.rarekidscan.com/training-and...

RareKids-CAN Open Call: Sponsored American Society for Gene & Cell Therapy - Attendance for Pediatric ATMP Clinical Trial Professionals  — RareKids-CAN Apply today to attend the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in May 2026.

Open Call! RareKids-CAN, in partnership with MICYRN, is offering sponsored virtual attendance to the 2026 @asgct.bsky.social Annual Meeting to support workforce development in pediatric ATMP clinical trials. 

Apply by Feb. 12 at 9am PST

Join us on Feb 4 for From Evidence to Access:
Advancing Pediatric Rare Disease Drug Availability in Canada.

Matthew McDonald & Douglas Clark will discuss the evolving role of HTA and reimbursement policy in #pediatric #raredisease care.

Register here: us02web.zoom.us/meeting/regi...

As 2025 wraps up, we’re reflecting on a year of growth at RareKids-CAN.  

Read our latest newsletter to learn more about this year’s growth, collaboration, and progress: mailchi.mp/micyrn/rarek...

Text on light purple background reads December 17, 2025, 1 to 2pm ET. Rare Methods Workshop Series, Biostatistical Methods for External Controls in Pediatric Rare Disease Clinical Trials. Register Today!

Join us for a virtual Rare Methods workshop. This session covers why external controls are used in pediatric rare-disease trials, key data sources, core biostatistical concepts for data integration, and more.
 
Register us02web.zoom.us/meeting/regi...
 
#RareDiseaseResearch #ClinicalTrials

RareKids-CAN joined SickKids Child Health Policy Accelerator & IMPaCT in Ottawa for Parliamentary Meetings with Minister Marjorie Michel to advance child health across Canada. Change happens when families, researchers & policymakers come together
#RareKidsCAN #ChildHealth #RareDisease #Collaboration

🎥 Join our Digital Storytelling Workshop on Oct 16 at 8 P.M. EST!

Learn how personal stories empower rare communities with Krystle Schofield—RareKids-CAN parent partner, B.C. mom, facilitator + photographer.

🔗 Register: zoom.us/meeting/regi...

#RareDisease #RareKidsCAN

Thank you to all who attended Family Engagement in Research Fundamentals! This session highlighted the fundamentals of patient and family engagement in research and how to strengthen engagement practices. For any questions or to learn more, please contact Sara and Alicia at rkengage@mcmaster.ca.

By embedding Indigenous voices, priorities, and knowledge into research, the Indigenous Community Engagement team is advancing cultural safety, trust, and equity in rare disease care and clinical trials. See how they’re driving change: tinyurl.com/2umpcfwt

Thank you to our #biostatistics team for hosting the 1st webinar in our new Rare Methods Workshop series, Bayesian Approaches to Pediatric Rare Disease Clinical Trials, and to our 80+ attendees! Missed it? No problem! Access the recording here: tinyurl.com/26h53pbe

#RareKidsCAN #RareDiseaseResearch

We're proud to partner with @impactrials.bsky.social to co-fund 2 training awards for projects focused on rare diseases with dedicated funding for Black and Indigenous applicants. Learn more and apply for a training award today: tinyurl.com/mrt5kd6j

#RareKidsCAN #RareDiseaseResearch #ClinicalTrials