You could be unlocking extra funds for rare disease research just by donating. Ask your HR department today and use the QR Code (or this link: med13l.org/donate/) to get started!
#CorporateMatching #MED13L #RAREDISEASERESEARCH #careaboutrare #med13lsyndrome #RareDisease
#CorporateMatching #MED13L #RAREDISEASERESEARCH #careaboutrare #med13lsyndrome #RareDisease
December 19, 2025 at 10:46 PM
You could be unlocking extra funds for rare disease research just by donating. Ask your HR department today and use the QR Code (or this link: med13l.org/donate/) to get started!
#CorporateMatching #MED13L #RAREDISEASERESEARCH #careaboutrare #med13lsyndrome #RareDisease
#CorporateMatching #MED13L #RAREDISEASERESEARCH #careaboutrare #med13lsyndrome #RareDisease
Published rare disease research? Share your story & win €2,000. New deadline Jan 5, 2026.
www.scientifyresearch.org/rare-disease...
Special thanks to our sponsors @kargerpublishers.bsky.social @lipotype.bsky.social
#RareDiseaseResearch #SciComm #RareDiseases #scientifyRESEARCH
www.scientifyresearch.org/rare-disease...
Special thanks to our sponsors @kargerpublishers.bsky.social @lipotype.bsky.social
#RareDiseaseResearch #SciComm #RareDiseases #scientifyRESEARCH
December 18, 2025 at 2:02 PM
Published rare disease research? Share your story & win €2,000. New deadline Jan 5, 2026.
www.scientifyresearch.org/rare-disease...
Special thanks to our sponsors @kargerpublishers.bsky.social @lipotype.bsky.social
#RareDiseaseResearch #SciComm #RareDiseases #scientifyRESEARCH
www.scientifyresearch.org/rare-disease...
Special thanks to our sponsors @kargerpublishers.bsky.social @lipotype.bsky.social
#RareDiseaseResearch #SciComm #RareDiseases #scientifyRESEARCH
Whoa… is it almost 2026 already? 😲 Before we welcome the new year, we’re taking a look back at the roadmap that got us here. Our 2024 Conference report is still a must-read. The power of collaboration is leading us into next year!
🔗 Full report: rarediseaseresearch....
🔗 Full report: rarediseaseresearch....
December 18, 2025 at 12:56 PM
Whoa… is it almost 2026 already? 😲 Before we welcome the new year, we’re taking a look back at the roadmap that got us here. Our 2024 Conference report is still a must-read. The power of collaboration is leading us into next year!
🔗 Full report: rarediseaseresearch....
🔗 Full report: rarediseaseresearch....
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
Learn about promising experimental treatments and clinical trials for hereditary angioedema
#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews
December 17, 2025 at 12:32 AM
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
Learn about promising experimental treatments and clinical trials for hereditary angioedema
#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews
New CF therapy update: https://bit.ly/44t9HhF
An inhaled investigational treatment showed encouraging safety results in a Phase 1 trial and remained in the lungs longer than expected.
#CysticFibrosis #CFResearch #ClinicalTrials #RareDiseaseResearch #CFNews #CysticFibrosisNewsToday #Bionews
An inhaled investigational treatment showed encouraging safety results in a Phase 1 trial and remained in the lungs longer than expected.
#CysticFibrosis #CFResearch #ClinicalTrials #RareDiseaseResearch #CFNews #CysticFibrosisNewsToday #Bionews
December 15, 2025 at 9:02 PM
New CF therapy update: https://bit.ly/44t9HhF
An inhaled investigational treatment showed encouraging safety results in a Phase 1 trial and remained in the lungs longer than expected.
#CysticFibrosis #CFResearch #ClinicalTrials #RareDiseaseResearch #CFNews #CysticFibrosisNewsToday #Bionews
An inhaled investigational treatment showed encouraging safety results in a Phase 1 trial and remained in the lungs longer than expected.
#CysticFibrosis #CFResearch #ClinicalTrials #RareDiseaseResearch #CFNews #CysticFibrosisNewsToday #Bionews
New ALS research insight: https://bit.ly/3MvvhvR
A new study suggests that patterns of genetic activity in blood may help detect ALS earlier and even offer clues about survival.
#ALS #ALSResearch #NeurodegenerativeDisease #RareDiseaseResearch #ALSNewsToday #Bionews
A new study suggests that patterns of genetic activity in blood may help detect ALS earlier and even offer clues about survival.
#ALS #ALSResearch #NeurodegenerativeDisease #RareDiseaseResearch #ALSNewsToday #Bionews
December 15, 2025 at 8:12 PM
New ALS research insight: https://bit.ly/3MvvhvR
A new study suggests that patterns of genetic activity in blood may help detect ALS earlier and even offer clues about survival.
#ALS #ALSResearch #NeurodegenerativeDisease #RareDiseaseResearch #ALSNewsToday #Bionews
A new study suggests that patterns of genetic activity in blood may help detect ALS earlier and even offer clues about survival.
#ALS #ALSResearch #NeurodegenerativeDisease #RareDiseaseResearch #ALSNewsToday #Bionews
On Dec. 11, our colleagues at @cpathinstitute.bsky.social are hosting a webinar recognizing six years of success in rare disease #DataSharing with the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP). Register today at: c-path-org.zoom.us/webinar/regi... #RareDiseaseResearch
December 4, 2025 at 4:00 PM
On Dec. 11, our colleagues at @cpathinstitute.bsky.social are hosting a webinar recognizing six years of success in rare disease #DataSharing with the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP). Register today at: c-path-org.zoom.us/webinar/regi... #RareDiseaseResearch
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
Register us02web.zoom.us/meeting/regi...
#RareDiseaseResearch #ClinicalTrials
December 4, 2025 at 12:52 PM
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
Register us02web.zoom.us/meeting/regi...
#RareDiseaseResearch #ClinicalTrials
Why communication is important full talk here: youtu.be/rzF1USN2ekg
Support evidence-based research and help families gain answers: give.rarevillage.org/campaign/697...
#GivingTuesday #SupportScience #SpeechResearch #LanguageResearch #RareDiseaseResearch #CACNA1C
Support evidence-based research and help families gain answers: give.rarevillage.org/campaign/697...
#GivingTuesday #SupportScience #SpeechResearch #LanguageResearch #RareDiseaseResearch #CACNA1C
December 2, 2025 at 2:07 PM
Why communication is important full talk here: youtu.be/rzF1USN2ekg
Support evidence-based research and help families gain answers: give.rarevillage.org/campaign/697...
#GivingTuesday #SupportScience #SpeechResearch #LanguageResearch #RareDiseaseResearch #CACNA1C
Support evidence-based research and help families gain answers: give.rarevillage.org/campaign/697...
#GivingTuesday #SupportScience #SpeechResearch #LanguageResearch #RareDiseaseResearch #CACNA1C
New article to read! New cases of δ-aminolevulinic acid dehydratase deficiency.
➡️ Three new cases of the world’s rarest porphyria (ALAD Deficiency).
➡️ Highlights lead-poisoning cases that mimic the disorder.
🔗 doi.org/10.1111/joim.70044
#RareDiseaseResearch #LeadToxicity
➡️ Three new cases of the world’s rarest porphyria (ALAD Deficiency).
➡️ Highlights lead-poisoning cases that mimic the disorder.
🔗 doi.org/10.1111/joim.70044
#RareDiseaseResearch #LeadToxicity
November 25, 2025 at 8:10 AM
New article to read! New cases of δ-aminolevulinic acid dehydratase deficiency.
➡️ Three new cases of the world’s rarest porphyria (ALAD Deficiency).
➡️ Highlights lead-poisoning cases that mimic the disorder.
🔗 doi.org/10.1111/joim.70044
#RareDiseaseResearch #LeadToxicity
➡️ Three new cases of the world’s rarest porphyria (ALAD Deficiency).
➡️ Highlights lead-poisoning cases that mimic the disorder.
🔗 doi.org/10.1111/joim.70044
#RareDiseaseResearch #LeadToxicity
Nearly all patients feel their disease is well-controlled, making it a promising new option for long-term HAE management.
#HAE #Angioedema #AngioedemaNews #RareDisease #RareDiseaseResearch #AngioedemaNews #Bionews
#HAE #Angioedema #AngioedemaNews #RareDisease #RareDiseaseResearch #AngioedemaNews #Bionews
November 24, 2025 at 3:02 PM
Nearly all patients feel their disease is well-controlled, making it a promising new option for long-term HAE management.
#HAE #Angioedema #AngioedemaNews #RareDisease #RareDiseaseResearch #AngioedemaNews #Bionews
#HAE #Angioedema #AngioedemaNews #RareDisease #RareDiseaseResearch #AngioedemaNews #Bionews
Every research participant brings us closer to answers families have waited for. If you or your child has a CAPRIN1 variant, we’d love to hear from you.
🔬 Sign up to participate in research today: caprin1foundation.org/participate-...
#RareDiseaseResearch #CAPRIN1 #ScienceForGood #GeneticResearch
🔬 Sign up to participate in research today: caprin1foundation.org/participate-...
#RareDiseaseResearch #CAPRIN1 #ScienceForGood #GeneticResearch
November 21, 2025 at 10:00 AM
Every research participant brings us closer to answers families have waited for. If you or your child has a CAPRIN1 variant, we’d love to hear from you.
🔬 Sign up to participate in research today: caprin1foundation.org/participate-...
#RareDiseaseResearch #CAPRIN1 #ScienceForGood #GeneticResearch
🔬 Sign up to participate in research today: caprin1foundation.org/participate-...
#RareDiseaseResearch #CAPRIN1 #ScienceForGood #GeneticResearch
⏰ The clock is ticking! Don't forget, the deadline for our Seed Funding is this coming Monday, Nov 25th.
Are you an emerging & early-career researcher looking to build data for a new Rare Disease intervention or trial? This is for you!
Apply: rarediseaseresearch....
Are you an emerging & early-career researcher looking to build data for a new Rare Disease intervention or trial? This is for you!
Apply: rarediseaseresearch....
November 20, 2025 at 11:17 AM
⏰ The clock is ticking! Don't forget, the deadline for our Seed Funding is this coming Monday, Nov 25th.
Are you an emerging & early-career researcher looking to build data for a new Rare Disease intervention or trial? This is for you!
Apply: rarediseaseresearch....
Are you an emerging & early-career researcher looking to build data for a new Rare Disease intervention or trial? This is for you!
Apply: rarediseaseresearch....
Tonight: Join 2025 International LAM Research Conference Co-Chairs, Dr. Henske and Dr. Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
November 18, 2025 at 3:00 PM
Tonight: Join 2025 International LAM Research Conference Co-Chairs, Dr. Henske and Dr. Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Tomorrow: Join 2025 International LAM Research Conference Co-Chairs, Dr. Henske and Dr. Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
November 17, 2025 at 9:00 PM
Tomorrow: Join 2025 International LAM Research Conference Co-Chairs, Dr. Henske and Dr. Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
⏳ Just one week to go! The deadline for our Seed Funding Application is next Monday, Nov 24th.
This is a fantastic opportunity to support early-career research. We're passionate about #PPI, so tell us your plans for collaboration!
Learn more & apply: rarediseaseresearch....
This is a fantastic opportunity to support early-career research. We're passionate about #PPI, so tell us your plans for collaboration!
Learn more & apply: rarediseaseresearch....
November 17, 2025 at 11:14 AM
⏳ Just one week to go! The deadline for our Seed Funding Application is next Monday, Nov 24th.
This is a fantastic opportunity to support early-career research. We're passionate about #PPI, so tell us your plans for collaboration!
Learn more & apply: rarediseaseresearch....
This is a fantastic opportunity to support early-career research. We're passionate about #PPI, so tell us your plans for collaboration!
Learn more & apply: rarediseaseresearch....
Join 2025 International LAM Research Conference Co-Chairs, Dr. Elizabeth Henske and Dr. Simon Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
November 15, 2025 at 11:00 PM
Join 2025 International LAM Research Conference Co-Chairs, Dr. Elizabeth Henske and Dr. Simon Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Join 2025 International LAM Research Conference Co-Chairs, Dr. Elizabeth Henske and Dr. Simon Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
November 11, 2025 at 9:15 PM
Join 2025 International LAM Research Conference Co-Chairs, Dr. Elizabeth Henske and Dr. Simon Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Join 2025 International LAM Research Conference Co-Chairs, Dr. Elizabeth Henske and Dr. Simon Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
November 4, 2025 at 9:15 PM
Join 2025 International LAM Research Conference Co-Chairs, Dr. Elizabeth Henske and Dr. Simon Johnson, as they share conference highlights, key takeaways, and what has them excited for the future of LAM research.
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
Register Now: https://www.tfaforms.com/5199266
#RareDiseaseResearch
An inspiring 2 days at the 2nd ERDERA General Assembly (Oct 30-31, 2025, Amsterdam)! We reviewed progress, identified challenges, and focused on improving data-sharing across borders.
#ERDERA #RareDiseaseResearch #DataSharing #Innovation
#ERDERA #RareDiseaseResearch #DataSharing #Innovation
November 4, 2025 at 11:00 AM
An inspiring 2 days at the 2nd ERDERA General Assembly (Oct 30-31, 2025, Amsterdam)! We reviewed progress, identified challenges, and focused on improving data-sharing across borders.
#ERDERA #RareDiseaseResearch #DataSharing #Innovation
#ERDERA #RareDiseaseResearch #DataSharing #Innovation
The venture philanthropy model is propelling research for Charcot-Marie-Tooth, a rare childhood genetic disease.
Wendy Paris(@wendyparis.bsky.social) reports: tinyurl.com/5n85c5vr
#RareDiseaseResearch #GeneTherapy #CMTResearchFoundation #ElpidaTherapeutics #CureCMT4J #VenturePhilanthropy
Wendy Paris(@wendyparis.bsky.social) reports: tinyurl.com/5n85c5vr
#RareDiseaseResearch #GeneTherapy #CMTResearchFoundation #ElpidaTherapeutics #CureCMT4J #VenturePhilanthropy
CMT Research Foundation Funds Landmark Gene Therapy Trial
The venture philanthropy model is propelling research for Charcot-Marie-Tooth, a rare childhood genetic disease.
tinyurl.com
October 29, 2025 at 7:00 PM
The venture philanthropy model is propelling research for Charcot-Marie-Tooth, a rare childhood genetic disease.
Wendy Paris(@wendyparis.bsky.social) reports: tinyurl.com/5n85c5vr
#RareDiseaseResearch #GeneTherapy #CMTResearchFoundation #ElpidaTherapeutics #CureCMT4J #VenturePhilanthropy
Wendy Paris(@wendyparis.bsky.social) reports: tinyurl.com/5n85c5vr
#RareDiseaseResearch #GeneTherapy #CMTResearchFoundation #ElpidaTherapeutics #CureCMT4J #VenturePhilanthropy
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
#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews
October 28, 2025 at 11:31 PM
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
#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews
In this week's "In the Thick of It," Allison Watson, CEO of Ring20 Research and Support UK CIO, shares crucial insights into Ring Chromosome 20 Syndrome (r(20)).
https://bit.ly/48KLWoc
#Ring20 #RareEpilepsy #PatientAdvocacy #RareDiseaseResearch #RingChromosome20
https://bit.ly/48KLWoc
#Ring20 #RareEpilepsy #PatientAdvocacy #RareDiseaseResearch #RingChromosome20
October 28, 2025 at 12:01 PM
In this week's "In the Thick of It," Allison Watson, CEO of Ring20 Research and Support UK CIO, shares crucial insights into Ring Chromosome 20 Syndrome (r(20)).
https://bit.ly/48KLWoc
#Ring20 #RareEpilepsy #PatientAdvocacy #RareDiseaseResearch #RingChromosome20
https://bit.ly/48KLWoc
#Ring20 #RareEpilepsy #PatientAdvocacy #RareDiseaseResearch #RingChromosome20
👉 Are you signed up for our newsletter? That’s where all the behind-the-scenes highlights, photos, and updates live! Don’t miss out - sign up today!
Link: teamtelomere.org/stay-connect...
#TeamTelomere #CocktailsAndChromosomes #RareDiseaseResearch #CommunityScience #MicroMeeting2025
Link: teamtelomere.org/stay-connect...
#TeamTelomere #CocktailsAndChromosomes #RareDiseaseResearch #CommunityScience #MicroMeeting2025
Stay Connected | Team Telomere
Every single day, Team Telomere is working to create a brighter future for those impacted by Telomere Biology Disorders. Stay up to date with research findings, community events, advocacy efforts,…
teamtelomere.org
October 23, 2025 at 6:56 PM
👉 Are you signed up for our newsletter? That’s where all the behind-the-scenes highlights, photos, and updates live! Don’t miss out - sign up today!
Link: teamtelomere.org/stay-connect...
#TeamTelomere #CocktailsAndChromosomes #RareDiseaseResearch #CommunityScience #MicroMeeting2025
Link: teamtelomere.org/stay-connect...
#TeamTelomere #CocktailsAndChromosomes #RareDiseaseResearch #CommunityScience #MicroMeeting2025
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
Learn about promising experimental treatments and clinical trials for hereditary angioedema
#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews
October 21, 2025 at 11:31 PM
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
Learn about promising experimental treatments and clinical trials for hereditary angioedema
#ClinicalTrials #ExperimentalTherapies #HAEResearch #RareDiseaseResearch #FutureOfMedicine #AngioedemaNews #Bionews