iiinova — where your digital self keeps evolving

RareKids-CAN @rarekidscan.bsky.social · 23/07/2026

Registration is now open for the Inaugural Canadian Gene & Cell Therapy Conference! This landmark event will bring together researchers, clinicians, manufacturers, industry leaders, regulators, trainees, and patient/family partners to shape the future of gene and cell therapy in Canada. cangct.ca

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RareKids-CAN @rarekidscan.bsky.social · 06/07/2026

This spring, RareKids-CAN supported Patient and Family Engagement Sub-Platform Lead Sara Pot's participation in the EURORDIS-Rare Diseases Europe Open Academy in Barcelona. Read her reflections here: lnkd.in/gg8Bimbm

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RareKids-CAN @rarekidscan.bsky.social · 01/07/2026

🍁 Happy Canada Day! 🍁 Working together across Canada, we're supporting research and clinical trials that help improve the future for children living with rare diseases. #CanadaDay #RareDisease #ClinicalTrials

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RareKids-CAN @rarekidscan.bsky.social · 24/06/2026

Join us on July 22nd for our next ATMP webinar: Delivering Gene Therapy Trials: Understanding Clinical Workflows, Operations, and the Patient Journey Register here: us02web.zoom.us/webinar/regi...

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RareKids-CAN @rarekidscan.bsky.social · 21/06/2026

On National Indigenous Peoples Day, we recognize and celebrate the histories, cultures, traditions, and contributions of First Nations, Inuit, and Métis Peoples. #NationalIndigenousPeoplesDay #NIPD2026

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RareKids-CAN @rarekidscan.bsky.social · 21/06/2026

This Father's Day, RareKids-CAN recognizes and celebrates the fathers and caregivers who support children and youth living with rare diseases.

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RareKids-CAN @rarekidscan.bsky.social · 16/06/2026

New publication from the RareKids-CAN Pharmacology Sub-Platform! The paper provides a guide to cell and gene therapies in pediatric rare disease research, covering clinical pharmacology, trial design, and regulatory considerations. Read more: www.rarekidscan.com/news/new-pap...

rarekidscan.com

New Paper Provides Practical Guide to Cell and Gene Therapies in Pediatric Rare Disease Research — RareKids-CAN

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 treatme...

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RareKids-CAN @rarekidscan.bsky.social · 15/06/2026

Global collaboration was a key theme at the World Orphan Drug Congress USA, where RareKids-CAN Nominated Principal Investigator and MICYRN Scientific Director Dr. Thierry Lacaze participated in a keynote panel exploring advances in rare disease research. www.rarekidscan.com/news/thierry...

rarekidscan.com

Thierry Lacaze Highlights the Importance of Global Collaboration at the World Orphan Drug Congress USA — RareKids-CAN

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 Bo...

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RareKids-CAN @rarekidscan.bsky.social · 11/06/2026

Last chance to register! Join us Monday, June 15 for a practical ATMP primer for clinical coordinators, nurses, and pharmacists working in pediatric rare disease trials. 📅 June 15 ⏰ 3 - 4 PM ET ➡️ Register here: us02web.zoom.us/webinar/regi... #PediatricRareDisease #RareDiseaseResearch

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RareKids-CAN @rarekidscan.bsky.social · 01/06/2026

LAST CHANCE TO REGISTER: Join us tomorrow (June 2nd) for From Evidence to Access: Do Lived and Clinical Experiences Shape HTA Decisions? RSVP: us02web.zoom.us/webinar/regi... #PediatricRareDisease #HTA #ClinicalResearch

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RareKids-CAN @rarekidscan.bsky.social · 29/05/2026

Improving access to pediatric rare disease #clinicaltrials requires more than research — it takes coordination, collaboration, and national readiness. Read our Q&A with Davy Eng, RareKids-CAN Clinical Trial Navigator here: www.rarekidscan.com/news/buildin... #RareDisease #PediatricResearch

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RareKids-CAN @rarekidscan.bsky.social · 28/05/2026

Join us June 15th for a practical introduction to Advanced Therapy Medicinal Products (ATMPs) for clinical coordinators, nurses, pharmacists, and research staff working in pediatric rare disease trials. Register now: us02web.zoom.us/webinar/regi... #pediatricraredisease #rarediseaseresearch

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RareKids-CAN @rarekidscan.bsky.social · 22/05/2026

As part of our #ClinicalTrialsDay Q&A series, RareKids-CAN Clinical Trial Navigator Daislyn Vidal shares insights on supporting families and helping improve access to #pediatricraredisease clinical trials. Read the Q&A here www.rarekidscan.com/news/connect...

"I see it as an honour to be able to directly help children suffering from rare diseases to get access to treatments in the form of trials which they normally would not get access to." - Daislyn Vidal Clinical Research Coordinator & RareKids-CAN Clinical Trial Navigator, University of Saskatchewan

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RareKids-CAN @rarekidscan.bsky.social · 19/05/2026

RareKids-CAN was proud to host our Canadian MEET & GREET at #ASGCT2026 in Boston and connect with colleagues and collaborators from across Canada advancing gene and cell therapy research. Stay tuned for details on our inaugural Canadian Gene & Cell Therapy conference coming in early 2027.

Network Director, Breanne Stewart, and Project Manager, Catherine Strandt at ASGCT Annual Meeting 2026

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RareKids-CAN @rarekidscan.bsky.social · 15/05/2026

Thank you to everyone who joined our Bill C-265 webinar yesterday. It sparked thoughtful conversation and shared commitment to advancing more efficient, patient-centred pathways to care. One way to take action now: support the campaign by sending a letter to your MP: politraq.ca/activations/...

politraq.ca

PoliTraQ — Track Your Advocacy Impact

Government affairs CRM for tracking stakeholder engagements, monitoring legislation, and measuring advocacy impact across Canadian jurisdictions.

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RareKids-CAN @rarekidscan.bsky.social · 14/05/2026

Join us June 2 for From Evidence to Access: Do Lived and Clinical Experiences Shape HTA Decisions? Register here: us02web.zoom.us/webinar/regi...

June 2 | 5pm ET | Virtual
From Evidence to Access: Do Lived and Clinical Experiences Shape HTA Decisions?

Chair: Dr. Maryam Oskoui, Sub-platform Lead, RareKids-CAN
Speakers:
Homira Osman, Vice President of Research Public Policy & Programs, Muscular Dystrophy Canada
Michelle Gibbens, Directory, Engagement, Canada's Drug Agency
Whitney Ayoub, Executive Director, ImmUnity Canada

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RareKids-CAN @rarekidscan.bsky.social · 12/05/2026

Don’t forget to register: Join us May 14th for a webinar about Bill C-265. Learn more: us02web.zoom.us/webinar/regi...

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RareKids-CAN @rarekidscan.bsky.social · 11/05/2026

Canadian attendees of the 2026 American Society of Gene & Cell Therapy Annual Meeting -- let's connect! 🇨🇦 Join us for a Canadian meet and greet to connect with colleagues, collaborators, and others from across Canada. Register here: luma.com/uz5kq3gp #ASGCT2026 #genetherapy #celltherapy

luma.com

MEET & GREET: Get together for Canadian Participants at ASGCT 2026 · Luma

Join fellow Canadian researchers, clinicians, industry, and partners attending ASGCT 2026 for an informal meet & greet. This is a great opportunity to connect,…

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RareKids-CAN @rarekidscan.bsky.social · 01/05/2026

Families are key partners in pediatric rare disease research. Through the FER Course, members of the RareKids-CAN community strengthened their skills and confidence to collaborate meaningfully. Read more: www.rarekidscan.com/news/strengt... #RareKidsCAN #FamilyEngagement #PatientPartnership

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RareKids-CAN @rarekidscan.bsky.social · 30/04/2026

Applications are now open for the Canadian Rare Disease Network (CRDN) RAREvolution Scholarship! This scholarship supports students aged 17 to 29 living with a diagnosed or suspected rare disease as they pursue post-secondary education in Canada. Learn more:: canadianrdn.ca/scholarship/

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RareKids-CAN @rarekidscan.bsky.social · 29/04/2026

Today is Undiagnosed Day, a moment to recognize the thousands of children’s and families still searching for answers. At RareKids-CAN, we know that diagnosis is more than a milestone, it’s the gateway to care, clinical trials, and treatments. #UndiagnosedDay #PediatricRareDisease #ClinicalTrials

White text on green background reads April 29th Undiagnosed Day 2026. An cartoon illustration of a doctor and child are next to the text. The child sits on a hospital bed as the doctor examines him. Floating around them are illustrations of paper work, DNA, hearts, and a magnifying glass

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RareKids-CAN @rarekidscan.bsky.social · 28/04/2026

Join us in supporting improvements to treatment access in Canada by writing to your MP in support of Bill C-265. politraq.ca/activations/... #BillC265 #HealthPolicy #HealthcareAccess #CanadianHealthcare #HealthAdvocacy

Text in yellow on purple background that reads "Write to your MP in suport of Bill C-265" Photo on the right of Canada's Parliment buildings centred on the Peace Tower and Library

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RareKids-CAN @rarekidscan.bsky.social · 23/04/2026

Join us May 14th to discuss Bill C-265 and what it could mean for access to treatment in Canada. Register here: us02web.zoom.us/webinar/regi... #BillC265 #HealthPolicy #HealthcareAccess #CanadianHealthcare #HealthAdvocacy

Join Us May 14th
Bill C-265: Improving Access to Essential Medications in Canada, featuring MP Marcus Powlowski

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RareKids-CAN @rarekidscan.bsky.social · 22/04/2026

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-...

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

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RareKids-CAN @rarekidscan.bsky.social · 14/04/2026

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

"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

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RareKids-CAN @rarekidscan.bsky.social · 07/04/2026

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

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

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RareKids-CAN @rarekidscan.bsky.social · 02/04/2026

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

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RareKids-CAN @rarekidscan.bsky.social · 31/03/2026

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

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.

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RareKids-CAN @rarekidscan.bsky.social · 30/03/2026

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

rarekidscan.com

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...

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RareKids-CAN @rarekidscan.bsky.social · 28/03/2026

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

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RareKids-CAN @rarekidscan.bsky.social · 24/03/2026

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

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.

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RareKids-CAN @rarekidscan.bsky.social · 23/03/2026

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

rarekidscan.com

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

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RareKids-CAN @rarekidscan.bsky.social · 16/03/2026

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

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RareKids-CAN @rarekidscan.bsky.social · 11/03/2026

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

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RareKids-CAN @rarekidscan.bsky.social · 10/03/2026

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

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RareKids-CAN @rarekidscan.bsky.social · 08/03/2026

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

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RareKids-CAN @rarekidscan.bsky.social · 05/03/2026

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

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Reposted by RareKids-CAN

McMaster University Department of Pediatrics @macupediatrics.bsky.social · 27/02/2026

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...

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RareKids-CAN @rarekidscan.bsky.social · 25/02/2026

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

rarekidscan.com

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RareKids-CAN @rarekidscan.bsky.social · 24/02/2026

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

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

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RareKids-CAN @rarekidscan.bsky.social · 10/02/2026

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

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

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RareKids-CAN @rarekidscan.bsky.social · 09/02/2026

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...

rarekidscan.com

Training and Webinars — RareKids-CAN

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RareKids-CAN @rarekidscan.bsky.social · 21/01/2026

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...

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RareKids-CAN @rarekidscan.bsky.social · 23/12/2025

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...

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RareKids-CAN @rarekidscan.bsky.social · 04/12/2025

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

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!

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