RareKids-CAN

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

Join us soon for this education session: tinyurl.com/3kz64jwj

Join us on Sept. 17, 2025, from 2-3pm ET for our next education session, led by our Patient and Family Engagement Facilitators. This session is open to everyone interested in strengthening engagement practices. Don't miss out: tinyurl.com/3kz64jwj

#RareKidsCAN #PatientEngagement #FamilyEngagement

Join RareKids-CAN for the first session in our new Rare Methods workshop series, “Bayesian Approaches to PRDCTs”, on September 16, 2025 from 1-2 PM ET. You can learn more about this session and register here: tr.ee/mH0nGPmnLe

#RareKidsCAN #RareDiseaseResearch

A research team at the University of Alberta is looking to hear feedback from parents of children with intellectual developmental disorders (IDD) on a new resource. Learn more at tinyurl.com/4bfmttyp

#PediatricRareDisease #RareDiseaseResearch #RareDiseaseClinicalTrials #RareDiseaseAdvocacy

The #RareKidsCAN Pharmacology Sub-Platform is organizing focus groups to better understand the experiences of patients and families when searching for information about medications & #ClinicalTrials for #PediatricRareDiseases. Learn how to get involved: tinyurl.com/2sdkbz2t

#RareDiseaseResearch

Researchers at Holland Bloorview are exploring whether sertraline can help reduce anxiety in children and youth with neurodevelopmental conditions. Find out more about how to get involved in this participation opportunity: tinyurl.com/4bfmttyp

#RareKidsCAN #PediatricRareDisease #ClinicalTrials

Earlier this year, Dr. Thierry Lacaze and Breanne Stewart attended the 6th Nordic Conference on Pediatric and Orphan Medicines in Helsinki, Finland. Learn more about how we're making an international impact: tinyurl.com/2uewjy89

#RareKidsCAN #PediatricRareDisease #RareDiseaseResearch

🎉 Exciting news! We're partnering with RareKids-CAN to co-fund two training awards for projects focused on rare diseases, including a dedicated award for Black or Indigenous applicants.

➡️ Learn more and apply: www.impactrials.ca/awardinfo

August 12 is #InternationalYouthDay! This year, we’re celebrating Simone, a dedicated #YouthPartner with #RareKidsCAN who’s making a difference in the #RareDiseaseCommunity. Discover Simone’s journey and how she became involved with our work: tinyurl.com/nhhhtchu

#LivingWithRare #PatientAdvocacy

We're excited to share that a CIHR Project Grant will support new work on rare disease patient registry recommendations, led by Dr. Andrea Tricco and PhD student Catherine Stratton—also a key member of the RareKids-CAN Registry Sub-Platform. Congrats, Catherine! Learn more: tinyurl.com/k65rhhf5

In June 2025, our Nominated Principal Investigator, Dr. Thierry Lacaze, and Network Director, Breanne Stewart, represented #RareKidsCAN at the in-person @erdera.bsky.social workshop held in Latvia. Read more about our exciting international collaboration: tinyurl.com/mr28fd7p

#PediatricRareDisease

We're excited to spotlight patient partner, Maureen Smith, who shares her story & calls for faster, better access to clinical trials in Canada. Thank you, Maureen, for pushing for change & better access! Read full story: tinyurl.com/39wcjtaj

#RareKidsCAN #RareDiseaseResearch #RareDiseaseAdvocacy

Join @impactrials.bsky.social for their upcoming webinar on July 21 from 12pm-1pm ET to learn more about current and future clinical trials in children from a European perspective. Register today: tinyurl.com/29yadvre

#RareKidsCAN #PediatricRareDisease #ClinicalTrials #RareDiseaseResearch

RareKids-CAN is proud to share the success story of a single patient study for a baby girl with a rare and severe immune disorder caused by a genetic mutation. Read more about this exciting development: tinyurl.com/mt9333hy

#RareKidsCAN #PediatricRareDisease #RareDiseaseResearch #GeneticDisorders

A research team at the University of Alberta is looking to hear feedback from parents of children with intellectual developmental disorders (IDD) on a new resource. Learn more at tinyurl.com/4bfmttyp

#PediatricRareDisease #RareDiseaseResearch #RareDiseaseClinicalTrials #RareDiseaseAdvocacy