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Learn about promising experimental treatments and clinical trials for hereditary angioedema

#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews

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

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

fatigue, weakness, swallowing difficulties, and the invisible labour of navigating complex care pathways.

🎙️ Link: youtu.be/bkk_okQ3mtk

#AutoimmuneDisease #RareDiseaseResearch #PatientPartners #TranslationalScience

🧵 End

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

Do organoids need to look like organs to model disease?
A new JIMD review argues that function may matter more than anatomy when studying inherited metabolic diseases.
Focus on the pathway - not the organ.
🔗 doi.org/10.1002/jimd...
#IMD #Organoids #RareDiseaseResearch

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

🔗To see the full poster visit: www.iamcharlene.com

🧡

#PatientLedResearch #RareDisease #RareDiseaseResearch #PosterPresentation #ResearchCommunity #RareCancer #IAmCharlene #RareGems #BionicWarrior #PPI #PPIE #PatientInvolvement

On #RareDiseaseDay, we honor the families and researchers advancing congenital disorder research. With 67% of Kids First data devoted to these conditions, shared data drives discovery.

#KidsFirstDRC #RareDiseaseResearch

Homepage Raising awareness for patients, families and carers around the world that are affected by rare diseases.

As a company committed to advancing rare disease research, we stand with those driving change in healthcare, diagnosis, and treatment. Join us in raising awareness and advocating for progress.

Learn more: rarediseaseday.org

#SupportScience #RareDiseaseResearch

On #RareDiseaseDay, let’s unite research and patient voices to advance science, uncover mechanisms, and drive innovation.

Together, academia can shape equity in diagnosis and therapy.

For more #RareDiseaseResearch, click here: www.sciencedirect.com/journal/gene...

#GenesAndDiseases #Science

On Rare Disease Day 2026, we recognise that advancing rare disease research is a collective effort, powered by people, strengthened by data, and built study by study.

https://perspectum.com/publications/?products=mrcp

#Perspectum #RareDiseaseDay #PSC #RareDiseaseResearch

Spatiotemporal analysis of human intestinal development at single-cell resolution Development of the human intestine is not well understood. Here, we link single-cell RNA sequencing and spatial transcriptomics to characterize intest…

📄 Key paper on single cell RNA sequencing and spatial transcriptomics in fetal intestine:
www.sciencedirect.com/science/arti...

#WIMMSpotlight #WIMMCommunity #PaediatricResearch #IntestinalImmunology #SpatialTranscriptomics #SingleCellGenomics #RareDiseaseResearch #MRCWIMM

Up to £100,000 for bold MND research.

In this Funder Perspective, @mndoddie5.bsky.social shares how they are accelerating innovative MND research in the UK.

🗓️ Deadline Feb 27
👉 Read here: lnkd.in/gvsU7giD

#ResearchFunding #MNDResearch #RareDiseaseResearch #FundingOpportunity #FunderPerspective

#26for26 #TeamTelomere #TelomereBiologyDisorders #DyskeratosisCongenita #RareDiseaseMonth #ResearchSeason #RareDiseaseCommunity #RareDiseaseResearch

New research shows that ACTA2 smooth muscle dysfunction leads to baseline failure of cerebrovascular reserve, reducing cerebral blood flow and increasing vulnerability to brain injury www.biorxiv.org/content/10.6... @biorxiv-neursci.bsky.social #MSMDS

#ACTA2 #MSMDS #BrainHealth #RareDiseaseResearch

Katie Runs for Rare & Rural I’m honored to be running in the 2026 United Airlines NYC Half on behalf of the EveryLife Foundation for Rare Diseases. This race is more than a personal challenge - it’s a commitment to the 30…

Link to donate: teamtelomere.networkforgood.com/projects/294...

#26for26 #TeamTelomere #ResearchSeason #RareDiseaseMonth #TelomereBiologyDisorders #RareDiseaseResearch #LegacyThroughResearch #HopeThroughScience #AdvocacyInAction #ResearchMatters #Bereavement #TransformationalResearch #Liver #BMF

Stay up to date on promising experimental treatments and clinical trials for hereditary angioedema: https://bit.ly/43PupIn

#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews

Katie Runs for Rare & Rural I’m honored to be running in the 2026 United Airlines NYC Half on behalf of the EveryLife Foundation for Rare Diseases. This race is more than a personal challenge - it’s a commitment to the 30…

💜🩷💚

Link to donate: teamtelomere.networkforgood.com/projects/294...

#26for26 #TeamTelomere #ResearchSeason #TelomereBiologyDisorders #RareDiseaseResearch #RareDiseaseMonth #AdvocacyInAction #DyskeratosisCongenita #BMF #Liver #PF #HopeThroughResearch #ResearchMatters

New Wins for Rare Disease Research, Funded by Philanthropy Private donors and advocates are crucial in the fight to keep rare disease research, and patients, alive.

Private donors and advocates are crucial in the fight to keep rare disease research, and patients, alive.

Wendy Paris(@wendyparis.bsky.social) Reports: tinyurl.com/y7yvdeht

#RareDiseaseResearch #PediatricCancer #DIPG #MedicalPhilanthropy #PatientAdvocacy #FamilyFoundations #DiseaseResearchFunding

Stay up to date: https://bit.ly/43PupIn

Learn about promising experimental treatments and clinical trials for hereditary angioedema

#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews

After losing her daughter to #SCN2A complications, Angie Weaver donates her daughter’s brain for #RareDiseaseResearch. This vulnerable story explores grief, love & what it means to make an ultimate gift in the hope of helping other families. 🎧 https://ow.ly/jzBM50YawZe

"New in the ALS News: Scientists lab-grow motor neurons mimicking specialized nerve cells lost in ALS."

Discover the new science: https://bit.ly/4agjgCx

Researchers have found a way to grow highly specialized motor neurons in the lab that closely resemble the cells lost in ALS.

#ALS #ALSNewsToday #Bionews #ALSAwareness #MotorNeuronDisease #RareDiseaseResearch

The Latest CF News: CMTX-101 therapy helps clear bacterial lung infections, well tolerated in early trial.

Research to watch: https://bit.ly/4szOltb

An experimental antibody therapy is being studied for its potential to help clear stubborn lung infections in cystic fibrosis.

#CysticFibrosis #CFResearch #ClinicalTrials #LungHealth #CFScience #RareDiseaseResearch #CysticFibrosisNewsToday #Bionews

Join Neil Ward and Dr. Mohammed Uddin for the PacBio workshop at #FOG2026 on Jan 28. Learn how HiFi long-read sequencing is advancing #rarediseaseresearch, helping to improve diagnostic findings and unlock insights from complex genomic regions.

Full programme details: bit.ly/3MAm7y1

Achieve unmatched precision with our #targetedresequencing services. Whether using gene panels or custom designs, gain valuable insights for research in oncology, #rarediseaseresearch, and more.

Learn how we support your breakthroughs: https://bit.ly/3ZYOoSY

#Genomics #CancerResearch #NGS

Arginase Deficiency Market Size, Share, Trends, Report 2035 Arginase Deficiency Market growth is projected to reach USD 1.466 Billion, at 9.72% CAGR by driving industry size, share, top company analysis, segments research, trends and forecast report 2025 to 20...

🧬 Arginase Deficiency research is advancing therapies for rare metabolic disorders. 💡📊 www.marketresearchfuture.com/reports/argi... #GeneticDisorders #RareDiseaseResearch #Biotech

Still thinking about it?

We’ve extended the deadline for the Science Communication Award to Monday, Jan 5

🔗 www.scientifyresearch.org/rare-disease...

Special thanks to our sponsors
@kargerpublishers.bsky.social @lipotype.bsky.social

#RareDisease #rareDiseaseResearch #ScienceCommunication

Stay up to date on promising experimental treatments and clinical trials for hereditary angioedema: https://bit.ly/43PupIn

#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews

🧬 Genetic testing can unlock answers about CAPRIN1

If you or a loved one has a suspected CAPRIN1 variant, consider genetic testing which will help researchers move closer to treatments for people worldwide.

Learn more: caprin1foundation.org/participate-...

#GeneticTesting #RareDiseaseResearch