#rarediseases — Public Fediverse posts

Dangerous tick-borne virus spreading to more Americans, experts warn

NEWYou can now listen to Fox News articles! A rare and potentially fatal tick-borne illness currently spreading across…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Health #infectiousdisease #Insects #Lifestyle #outbreaks #outdoorlivingrealestatelifestyle #rarediseases #Viruses
https://www.newsbeep.com/us/733680/

Dangerous tick-borne virus spreading to more Americans, experts warn

NEWYou can now listen to Fox News articles! A rare and potentially fatal tick-borne illness currently spreading across…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Health #infectiousdisease #Insects #Lifestyle #outbreaks #outdoorlivingrealestatelifestyle #rarediseases #Viruses
https://www.newsbeep.com/us/733680/

Incyte (INCY) Wins EU Backing For Opzelura As Pipeline Data Builds Across Four Drugs

Get insights on thousands of stocks from the global community of over 7 million individual investors at Simply…
#Europe #EU #atopicdermatitis #EuropeanCommission #EuropeanUnion #globalcommunity #Incyte #individualinvestors #myeloproliferativeneoplasms #Opzelura #rareconditions #rarediseases #regulatoryupdates #treatmentguidelines
https://www.europesays.com/europe/80625/

https://www.europesays.com/ch/82235/ Novartis dystrophy data tee up regulatory talks on Avidity drug #biotech #ClinicalData #FierceBiotechHomepage #MuscularDystrophy #Novartis #RareDiseases #siRNA

#VeteransAdministration #RareDiseases #Military

From Stars and Stripes: Veteran with rare muscle-wasting disease seeks VA recognition

https://www.stripes.com/veterans/2026-05-29/veteran-rare-disease-va-recognition-21816111.html

#VeteransAdministration #RareDiseases #Military

From Stars and Stripes: Veteran with rare muscle-wasting disease seeks VA recognition

https://www.stripes.com/veterans/2026-05-29/veteran-rare-disease-va-recognition-21816111.html

#VeteransAdministration #RareDiseases #Military

From Stars and Stripes: Veteran with rare muscle-wasting disease seeks VA recognition

https://www.stripes.com/veterans/2026-05-29/veteran-rare-disease-va-recognition-21816111.html

𝗥𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 𝘁𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝗱𝗲𝗺𝗮𝗻𝗱 𝗮𝗰𝗿𝗼𝘀𝘀 𝘁𝗵𝗲 𝗠𝗶𝗱𝗱𝗹𝗲 𝗘𝗮𝘀𝘁 𝗶𝘀 𝗲𝗻𝘁𝗲𝗿𝗶𝗻𝗴 𝗮 𝗵𝗶𝗴𝗵 𝗴𝗿𝗼𝘄𝘁𝗵 𝗵𝗲𝗮𝗹𝘁𝗵𝗰𝗮𝗿𝗲 𝗽𝗵𝗮𝘀𝗲!
The 𝗠𝗶𝗱𝗱𝗹𝗲 𝗘𝗮𝘀𝘁 𝗥𝗮𝗿𝗲 𝗗𝗶𝘀𝗲𝗮𝘀𝗲𝘀 𝗧𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝗠𝗮𝗿𝗸𝗲𝘁 is expanding as governments improve healthcare access, diagnostics, and specialty drug availability. Rising awareness and advancements in personalized medicine are reshaping patient care outcomes.
𝗥𝗲𝗮𝗱 𝗺𝗼𝗿𝗲: https://www.kenresearch.com/middle-east-rare-diseases-treatment-market
#Healthcare #RareDiseases #MiddleEast #PharmaIndustry

siRNA Duplexes Market in Germany | Report – IndexBox

#Germany #DE #Europe #EU #Europa #BioinformaticsforsiRNAdesign&off-targetprediction #biopharmamarketreport #Chemicalmodificationchemistries #forecast #Genefunctionstudies #High-throughputgeneticscreening #High-throughputpurification&QC(HPLC #marketanalysis #MS) #rarediseases) #siRNAduplexes #Solid-phaseoligonucleotidesynthesis #Targetidentification/validation #Therapeuticcandidatedevelopment(oncology
https://www.europesays.com/germany/11517/

This is the first step to do the amazing at CSL Plasma. Your help is essential to create life-saving medicines for patients in need. Use my link and we both get rewarded.
https://rewards.cslplasma.com/referral/referral-unique-code/eyJkb25vcklEIjoiMDBFQzNTOSIsImNvZGUiOiI5OU1GN0JNS1ZEIiwicmVnaW9uU2hvcnROYW1lIjoidXMifQ

#CSL #Donate #DonatePlasma #PlasmaDonation
#PlasmaDonor #SaveLives #BloodPlasma #GivePlasma #BeAHero
#CommunitySupport #MakeADifference #HelpOthers
#PayItForward #PlasmaTherapy
#MedicalResearch
#ChronicIllnessSupport #Healthcare
#RareDiseases #SideHustle #ExtraCash #PaidToSaveLives

This is the first step to do the amazing at CSL Plasma. Your help is essential to create life-saving medicines for patients in need. Use my link and we both get rewarded.
https://rewards.cslplasma.com/referral/referral-unique-code/eyJkb25vcklEIjoiMDBFQzNTOSIsImNvZGUiOiI5OU1GN0JNS1ZEIiwicmVnaW9uU2hvcnROYW1lIjoidXMifQ

#CSL #Donate #DonatePlasma #PlasmaDonation
#PlasmaDonor #SaveLives #BloodPlasma #GivePlasma #BeAHero
#CommunitySupport #MakeADifference #HelpOthers
#PayItForward #PlasmaTherapy
#MedicalResearch
#ChronicIllnessSupport #Healthcare
#RareDiseases #SideHustle #ExtraCash #PaidToSaveLives

This is the first step to do the amazing at CSL Plasma. Your help is essential to create life-saving medicines for patients in need. Use my link and we both get rewarded.
https://rewards.cslplasma.com/referral/referral-unique-code/eyJkb25vcklEIjoiMDBFQzNTOSIsImNvZGUiOiI5OU1GN0JNS1ZEIiwicmVnaW9uU2hvcnROYW1lIjoidXMifQ

#CSL #Donate #DonatePlasma #PlasmaDonation
#PlasmaDonor #SaveLives #BloodPlasma #GivePlasma #BeAHero
#CommunitySupport #MakeADifference #HelpOthers
#PayItForward #PlasmaTherapy
#MedicalResearch
#ChronicIllnessSupport #Healthcare
#RareDiseases #SideHustle #ExtraCash #PaidToSaveLives

This is the first step to do the amazing at CSL Plasma. Your help is essential to create life-saving medicines for patients in need. Use my link and we both get rewarded.
https://rewards.cslplasma.com/referral/referral-unique-code/eyJkb25vcklEIjoiMDBFQzNTOSIsImNvZGUiOiI5OU1GN0JNS1ZEIiwicmVnaW9uU2hvcnROYW1lIjoidXMifQ

#CSL #Donate #DonatePlasma #PlasmaDonation
#PlasmaDonor #SaveLives #BloodPlasma #GivePlasma #BeAHero
#CommunitySupport #MakeADifference #HelpOthers
#PayItForward #PlasmaTherapy
#MedicalResearch
#ChronicIllnessSupport #Healthcare
#RareDiseases #SideHustle #ExtraCash #PaidToSaveLives

This is the first step to do the amazing at CSL Plasma. Your help is essential to create life-saving medicines for patients in need. Use my link and we both get rewarded.
https://rewards.cslplasma.com/referral/referral-unique-code/eyJkb25vcklEIjoiMDBFQzNTOSIsImNvZGUiOiI5OU1GN0JNS1ZEIiwicmVnaW9uU2hvcnROYW1lIjoidXMifQ

#CSL #Donate #DonatePlasma #PlasmaDonation
#PlasmaDonor #SaveLives #BloodPlasma #GivePlasma #BeAHero
#CommunitySupport #MakeADifference #HelpOthers
#PayItForward #PlasmaTherapy
#MedicalResearch
#ChronicIllnessSupport #Healthcare
#RareDiseases #SideHustle #ExtraCash #PaidToSaveLives

Wir starten in die letzte Inhalationswoche.

Dank den Frühlingsferien müssen wir wenigstens nicht schon 6 oder 7 Uhr anfangen. Also schaffen wir das doch locker 💪😆

#inhalation #antibiotikum #ataxiateleangiectasia #lungenkrankheit #rarediseases

Wir starten in die letzte Inhalationswoche.

Dank den Frühlingsferien müssen wir wenigstens nicht schon 6 oder 7 Uhr anfangen. Also schaffen wir das doch locker 💪😆

#inhalation #antibiotikum #ataxiateleangiectasia #lungenkrankheit #rarediseases

Wir starten in die letzte Inhalationswoche.

Dank den Frühlingsferien müssen wir wenigstens nicht schon 6 oder 7 Uhr anfangen. Also schaffen wir das doch locker 💪😆

#inhalation #antibiotikum #ataxiateleangiectasia #lungenkrankheit #rarediseases

I’ll attend the #EuroNDD Workshop in Warsaw in April. Anyone around the Fediverse going there as well? —> let’s connect!

I’m looking forward to present our work at https://www.findme2care.de AND hear about all the other projects.

Also: I’ll be co-hosting an educational session and roundtable discussion titled „Building Patient Registries under the GDPR – The Good, the Bad and the Ugly“ —> there are a few seats left for conference attendees!

#humangenetics #genetics #RareDiseases #RareDisease #ERN #ERNIthaca #patientregistry

I’ll attend the #EuroNDD Workshop in Warsaw in April. Anyone around the Fediverse going there as well? —> let’s connect!

I’m looking forward to present our work at https://www.findme2care.de AND hear about all the other projects.

Also: I’ll be co-hosting an educational session and roundtable discussion titled „Building Patient Registries under the GDPR – The Good, the Bad and the Ugly“ —> there are a few seats left for conference attendees!

#humangenetics #genetics #RareDiseases #RareDisease #ERN #ERNIthaca #patientregistry

https://www.europesays.com/ch/35861/ Roche stops work on experimental SMA drug #MuscularDystrophy #RareDiseases #Roche #ScholarRock #SMAPatients #SpinalMuscularAtrophy

💡 A genuine surprise: The active ingredient in #Viagra, sildenafil, helps treat a rare genetic disorder! This unexpected finding has now been uncovered by researchers at #CharitéBerlin. The drug showed positive effects on the course of the disease in six patients with #Leigh syndrome. More on the promising results:

👉 https://www.charite.de/en/service/press_reports/artikel/detail/active_ingredient_of_viagra_helps_treat_rare_genetic_disease

#medicine #science #research #MedMastodon #RareDiseases

💡 A genuine surprise: The active ingredient in #Viagra, sildenafil, helps treat a rare genetic disorder! This unexpected finding has now been uncovered by researchers at #CharitéBerlin. The drug showed positive effects on the course of the disease in six patients with #Leigh syndrome. More on the promising results:

👉 https://www.charite.de/en/service/press_reports/artikel/detail/active_ingredient_of_viagra_helps_treat_rare_genetic_disease

#medicine #science #research #MedMastodon #RareDiseases

Gene therapy revolutionizes medicine by correcting defective genes and treating inherited diseases, cancers, and rare disorders with advanced biotechnology innovations.
#GeneTherapy #Biotechnology #GeneticEngineering #MedicalInnovation #PrecisionMedicine #CancerResearch #RareDiseases #BiotechStudents #LifeSciences #BioResire

Gene therapy revolutionizes medicine by correcting defective genes and treating inherited diseases, cancers, and rare disorders with advanced biotechnology innovations.
#GeneTherapy #Biotechnology #GeneticEngineering #MedicalInnovation #PrecisionMedicine #CancerResearch #RareDiseases #BiotechStudents #LifeSciences #BioResire

💪 Could one molecular “typo” break the balance between strength and flexibility in our muscles?

🔗 Dynamical features of smooth muscle actin pathological mutants: The arginine-257(258)-Cysteine cases. Computational and Structural Biotechnology Journal, DOI: https://doi.org/10.1016/j.csbj.2025.02.010

📚 CSBJ: https://www.csbj.org/

#ComputationalBiology #MolecularDynamics #ProteinScience #Actin #RareDiseases #PrecisionMedicine #Biophysics #CryoEM #Bioinformatics #DrugDiscovery #VisceralMyopathy #AorticAneurysm

💪 Could one molecular “typo” break the balance between strength and flexibility in our muscles?

🔗 Dynamical features of smooth muscle actin pathological mutants: The arginine-257(258)-Cysteine cases. Computational and Structural Biotechnology Journal, DOI: https://doi.org/10.1016/j.csbj.2025.02.010

📚 CSBJ: https://www.csbj.org/

#ComputationalBiology #MolecularDynamics #ProteinScience #Actin #RareDiseases #PrecisionMedicine #Biophysics #CryoEM #Bioinformatics #DrugDiscovery #VisceralMyopathy #AorticAneurysm

💪 Could one molecular “typo” break the balance between strength and flexibility in our muscles?

🔗 Dynamical features of smooth muscle actin pathological mutants: The arginine-257(258)-Cysteine cases. Computational and Structural Biotechnology Journal, DOI: https://doi.org/10.1016/j.csbj.2025.02.010

📚 CSBJ: https://www.csbj.org/

#ComputationalBiology #MolecularDynamics #ProteinScience #Actin #RareDiseases #PrecisionMedicine #Biophysics #CryoEM #Bioinformatics #DrugDiscovery #VisceralMyopathy #AorticAneurysm

💪 Could one molecular “typo” break the balance between strength and flexibility in our muscles?

🔗 Dynamical features of smooth muscle actin pathological mutants: The arginine-257(258)-Cysteine cases. Computational and Structural Biotechnology Journal, DOI: https://doi.org/10.1016/j.csbj.2025.02.010

📚 CSBJ: https://www.csbj.org/

#ComputationalBiology #MolecularDynamics #ProteinScience #Actin #RareDiseases #PrecisionMedicine #Biophysics #CryoEM #Bioinformatics #DrugDiscovery #VisceralMyopathy #AorticAneurysm

💪 Could one molecular “typo” break the balance between strength and flexibility in our muscles?

🔗 Dynamical features of smooth muscle actin pathological mutants: The arginine-257(258)-Cysteine cases. Computational and Structural Biotechnology Journal, DOI: https://doi.org/10.1016/j.csbj.2025.02.010

📚 CSBJ: https://www.csbj.org/

#ComputationalBiology #MolecularDynamics #ProteinScience #Actin #RareDiseases #PrecisionMedicine #Biophysics #CryoEM #Bioinformatics #DrugDiscovery #VisceralMyopathy #AorticAneurysm

Finally, this work would not have been possible without the input and work from all my co-authors from UK, France, Italy and USA. I am grateful that I could be part of this journee and want to thank also the #FoundationForPraderWilliResearch (#FPWR) for their generous funding! I hope that this research may at some day contribute to improve the life of patients with #PraderWilliSyndrome (#PWS) or other #RareDiseases.

#TranslationalResearch #INFRAFRONTIER #IMPC

Finally, this work would not have been possible without the input and work from all my co-authors from UK, France, Italy and USA. I am grateful that I could be part of this journee and want to thank also the #FoundationForPraderWilliResearch (#FPWR) for their generous funding! I hope that this research may at some day contribute to improve the life of patients with #PraderWilliSyndrome (#PWS) or other #RareDiseases.

#TranslationalResearch #INFRAFRONTIER #IMPC

Only four people in Australia have been diagnosed with rare genetic disease CHOPS

Isla St…
#NewsBeep #News #Genetics #AFF4 #AU #Australia #CHOPS #CHOPSaustralia #CHOPSsyndrome #chopssyndromeglobal #Cognitiveimpairmentandcoarsefacialfeatures #CorneliadeLange #CorneliaDeLangeSyndrome #extremelyrarediseases #extremelyraregeneticdisorders #FondazioneCHOPS #geneticdiseases #Health #IanKrantz #KosukeIzumi #RareDiseases #Science #shortstatureandskeletalabnormalities
https://www.newsbeep.com/au/204356/

Only four people in Australia have been diagnosed with rare genetic disease CHOPS https://www.byteseu.com/1439518/ #AFF4 #Australia #CHOPS #CHOPSAustralia #CHOPSSyndrome #ChopsSyndromeGlobal #CognitiveImpairmentAndCoarseFacialFeatures #CorneliaDeLange #CorneliaDeLangeSyndrome #ExtremelyRareDiseases #ExtremelyRareGeneticDisorders #FondazioneCHOPS #GeneticDiseases #Health #IanKrantz #KosukeIzumi #RareDiseases #ShortStatureAndSkeletalAbnormalities

Developments in diagnostic and surgical techniques in children with sagittal suture craniosynostosis: a systematic review spanning the last 30 years | Orphanet Journal of Rare Diseases

Literature search and stu…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Genetics #Developmentofsurgicalinterventions #general #HumanGenetics #Literaturereview #Medicine/PublicHealth #Pharmacology/Toxicology #rarediseases #Sagittalsuturecraniosynostosis #Science
https://www.newsbeep.com/us/89436/

Developments in diagnostic and surgical techniques in children with sagittal suture craniosynostosis: a systematic review spanning the last 30 years | Orphanet Journal of Rare Diseases

Literature search and stu…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Genetics #Developmentofsurgicalinterventions #general #HumanGenetics #Literaturereview #Medicine/PublicHealth #Pharmacology/Toxicology #rarediseases #Sagittalsuturecraniosynostosis #Science
https://www.newsbeep.com/us/89436/

🧬 Happening today! Join us for a GHGA lecture by Prof. Rami Abou Jamra (University of Leipzig):
"Genome sequencing for rare disease diagnostics: uncovering hidden causal variants"
Learn how genome sequencing is advancing the diagnosis of rare diseases beyond exome analysis.

📅 May 21, 2025 | 🕒 4PM
🔗 Register here: https://dkfz-de.zoom.us/meeting/register/eE1w4zLpTFO4z2jcuUxbDg#/registration

Part of the Advances in Data-Driven Biomedicine series.
#Genomics #RareDiseases #GHGA #BiomedicalResearch #GenomeSequencing

📯 Just published! The February IHI newsletter at https://europa.eu/!cNp8tC
✅ How our projects are helping to make the #EHDS a reality
✅ Our MACUSTAR project on eye disease received a letter of support from the EMA
✅ AMR project COMBINE trained an #AI model to improve the selection of compounds that can fight multi-drug resistant bacteria
✅ We meet new IHI projects on #RareDiseases, greener drug manufacture & more patient friendly #BloodTests
#RareDiseaseDay #IHITransformingHealth #HorizonEU

📯 Just published! The February IHI newsletter at https://europa.eu/!cNp8tC
✅ How our projects are helping to make the #EHDS a reality
✅ Our MACUSTAR project on eye disease received a letter of support from the EMA
✅ AMR project COMBINE trained an #AI model to improve the selection of compounds that can fight multi-drug resistant bacteria
✅ We meet new IHI projects on #RareDiseases, greener drug manufacture & more patient friendly #BloodTests
#RareDiseaseDay #IHITransformingHealth #HorizonEU

Binance Labs ventures into DeSci with BIO Protocol investment - The decentralized science DAO supports healthcare efforts that might oth... - https://cointelegraph.com/news/binance-labs-invests-in-bio-protocol #decentralizedscience #healthsciencefunding #quantumbiology #rarediseases #binancelabs #bioprotocol #longevity #biotech #ip-nfts #biodao #desci

Binance Labs ventures into DeSci with BIO Protocol investment - The decentralized science DAO supports healthcare efforts that might oth... - https://cointelegraph.com/news/binance-labs-invests-in-bio-protocol #decentralizedscience #healthsciencefunding #quantumbiology #rarediseases #binancelabs #bioprotocol #longevity #biotech #ip-nfts #biodao #desci

Binance Labs ventures into DeSci with BIO Protocol investment - The decentralized science DAO supports healthcare efforts that might oth... - https://cointelegraph.com/news/binance-labs-invests-in-bio-protocol #decentralizedscience #healthsciencefunding #quantumbiology #rarediseases #binancelabs #bioprotocol #longevity #biotech #ip-nfts #biodao #desci

Binance Labs ventures into DeSci with BIO Protocol investment - The decentralized science DAO supports healthcare efforts that might oth... - https://cointelegraph.com/news/binance-labs-invests-in-bio-protocol #decentralizedscience #healthsciencefunding #quantumbiology #rarediseases #binancelabs #bioprotocol #longevity #biotech #ip-nfts #biodao #desci

Arrived!

#Cilia2024 about to start😊👍

@cilia @cellbio #cilia #rarediseases #ciliopathies #centrosome

👋 Say hello to our new IHI project, PaLaDIn! Their goal is to develop a platform that collects innovative, real-world data #RWD from people with #RareDiseases. 👉 More: https://www.ihi.europa.eu/news-events/newsroom/paladin-project-puts-rare-disease-patients-centre-data-collection-platform #IHITransformingHealth #EUResearch #HealthResearch #Health #Research #EU #Data

Hola Titánicos, el mes de Febrero, es el mes de las Enfermedades Raras, y este año se celebra bajo el lema : "Comparte tus colores"

https://somosdisca.es/mes-de-las-enfermedades-raras-2024/

#febrero #mesdelasnefermedadesraras
#febreroraro #enfermedadrara #enfermedadesraras #Disability #disabilities #RareDiseases discapacidad #rare_diseases #RareDiseasesday #salud #enfermedad #visibilidad #cuidados #investigacion #diferente #cebra #titanicos #titan #gentetitanica #somosdisca

Hola Titánicos, el mes de Febrero, es el mes de las Enfermedades Raras, y este año se celebra bajo el lema : "Comparte tus colores"

https://somosdisca.es/mes-de-las-enfermedades-raras-2024/

#febrero #mesdelasnefermedadesraras
#febreroraro #enfermedadrara #enfermedadesraras #Disability #disabilities #RareDiseases discapacidad #rare_diseases #RareDiseasesday #salud #enfermedad #visibilidad #cuidados #investigacion #diferente #cebra #titanicos #titan #gentetitanica #somosdisca

📣 📣 📣 There are less than nine months remaining until Cilia2024, happening from September 10th to 13th in Dublin! 🇮🇪 :happy:

Remember to mark your calendars! What an amazing lineup of programs and speakers!

For more information, visit http://cilia2024.ie/

Registration opens in February.

@cilia @cellbiology @cellbio
#CellBiology #DevelopmentalBiology #DevBio #Nephrology #Ciliopathies #cilia #Science #Conference #RareDiseases #Kidney #Biology #Flagella #PCD #ADPKD #BBS #NPHP #CKD #PKD

German researchers welcome national, EU support for gene therapy research https://www.euractiv.com/section/health-consumers/news/german-researchers-welcome-national-and-eu-support-for-gene-therapy-research/?utm_source=dlvr.it&utm_medium=mastodon #genetherapies #Germany #rarediseases

German researchers welcome national, EU support for gene therapy research https://www.euractiv.com/section/health-consumers/news/german-researchers-welcome-national-and-eu-support-for-gene-therapy-research/?utm_source=dlvr.it&utm_medium=mastodon #genetherapies #Germany #rarediseases

EU needs to step up to unleash gene therapies potential, stakeholders say https://www.euractiv.com/section/health-consumers/news/eu-needs-to-step-up-to-unleash-gene-therapies-potential-stakeholders-say/?utm_source=dlvr.it&utm_medium=mastodon #ATMPs #genetherapies #rarediseases