#rarediseases — Public Fediverse posts

Soligenix reports positive Phase 2 trial results for SGX945 in treating Behçet's disease, offering new hope for patients with rare inflammatory condition #MedicalResearch #RareDiseases

Soligenix reports positive Phase 2 trial results for SGX945 in treating Behçet's disease, offering new hope for patients with rare inflammatory condition #MedicalResearch #RareDiseases

Soligenix reports positive Phase 2 trial results for SGX945 in treating Behçet's disease, offering new hope for patients with rare inflammatory condition #MedicalResearch #RareDiseases

Soligenix reports positive Phase 2 trial results for SGX945 in treating Behçet's disease, offering new hope for patients with rare inflammatory condition #MedicalResearch #RareDiseases

Soligenix reports positive Phase 2 trial results for SGX945 in treating Behçet's disease, offering new hope for patients with rare inflammatory condition #MedicalResearch #RareDiseases

Soligenix reports positive Phase 2 trial results for SGX945 in treating Behçet's disease, offering new hope for patients with rare inflammatory condition #MedicalResearch #RareDiseases

Gotta try to get back #sleep. I ache all over right now Nurse gave me atarax and norco tonight to help. Good night to all. 1231 am in the PNW. #Insomnis&Pain sucks. #ChronicIllness #ChronicPain #RareDiseases I am a #zebra

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

The four types of dementia most people don’t know exist
#Dementia #HealthAwareness #BrainHealth #Alzheimers #Neurology #MedicalEducation #PublicHealth #RareDiseases #MentalHealth #Healthcare #Awareness #Science #Caregiving #Aging
https://the-14.com/the-four-types-of-dementia-most-people-dont-know-exist/

The four types of dementia most people don’t know exist
#Dementia #HealthAwareness #BrainHealth #Alzheimers #Neurology #MedicalEducation #PublicHealth #RareDiseases #MentalHealth #Healthcare #Awareness #Science #Caregiving #Aging
https://the-14.com/the-four-types-of-dementia-most-people-dont-know-exist/

The four types of dementia most people don’t know exist
#Dementia #HealthAwareness #BrainHealth #Alzheimers #Neurology #MedicalEducation #PublicHealth #RareDiseases #MentalHealth #Healthcare #Awareness #Science #Caregiving #Aging
https://the-14.com/the-four-types-of-dementia-most-people-dont-know-exist/

The four types of dementia most people don’t know exist
#Dementia #HealthAwareness #BrainHealth #Alzheimers #Neurology #MedicalEducation #PublicHealth #RareDiseases #MentalHealth #Healthcare #Awareness #Science #Caregiving #Aging
https://the-14.com/the-four-types-of-dementia-most-people-dont-know-exist/

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

💡 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

February is Rare Disease Month, with Saturday 28 Feb being Rare Disease Day. To highlight this, Disease Models & Mechanisms is promoting its subject collection on rare disease research.

All articles are Open Access, so free to read and share for everyone: https://journals.biologists.com/dmm/collection/39/Rare-Disease-Translational-Research

#RareDiseases #DiseaseModels

RT by @EU_EESC: 🔴 EESC member @MilenaAngel23 #ReportingFromPlenary

1⃣ @EU_EESC backs European Life Science Strategy >@EUScienceInnov
2⃣ The 🇪🇺 needs a #RareDiseases action plan
3⃣ Research & technology are paramount for health

>@EESC_TEN

Click here & find out more 👉https://link.europa.eu/tWWqXg

🔴 EESC member @MilenaAngel23 #ReportingFromPlenary

1⃣ @EU_EESC backs European Life Science Strategy @EUScienceInnov
2⃣ The 🇪🇺 needs a #RareDiseases actio...
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https://nitter.net/EESC_PRESS/status/2024166292203171849#m

We need to do more for patients & their families!

We call for:
🧬holistic approach to #RareDiseases;
🧬stronger EU coordination in research & infrastructure;
🧬patients organisations and #EUCivilSociety involvement & participation in governance.

Opinion: http://link.europa.eu/tWWqXg
---
https://nitter.net/EU_EESC/status/2024156787927118024#m

Here's the original story: https://www.news-medical.net/news/20260110/AI-automates-sister-chromatid-exchange-counting-improving-diagnosis-of-Bloom-syndrome.aspx

#AI #MedicalAI #Healthcare #Tech #ArtificialIntelligence #RareDiseases

While CRISPR has opened doors for common genetic conditions, patients with rare diseases often miss out.

KJ's case showcases the potential for tailored therapies that could change the landscape, overcoming limitations in drug development for low-patient diseases.

#RareDiseases #Biotech

exemption to try to get care, but that usually ends up being a nightmare.

But maybe after the Nutcracker is solved and I'm feeling better, I will have the energy to keep pushing forward.

#NutcrackerSyndrome
#RareDiseases
#VascularCompressions

Soligenix advances rare disease therapies with innovative photodynamic and vaccine technologies, targeting critical unmet medical needs across multiple therapeutic areas. Breaking new ground in biopharmaceutical research. #RareDiseases #Biotech

Soligenix reports no safety concerns in Phase 3 FLASH2 trial for HyBryte, advancing treatment options for early-stage cutaneous T-cell lymphoma. Promising progress in rare disease therapeutics. #Biotech #RareDiseases

https://winbuzzer.com/2025/11/24/harvards-popeve-ai-model-identifies-123-novel-disease-genes-xcxwbn

Harvard’s popEVE AI Model Identifies 123 Novel Disease Genes

#AI #GoogleDeepMind #MedicalResearch #Genetics #RareDiseases #popEVE #AlphaMissense #HealthTech #Science #MachineLearning #Bioinformatics #Harvard

Soligenix advances rare disease treatments with promising clinical progress, maintaining strong safety profile and expanding therapeutic pipeline through 2026. Focused on innovative photodynamic and vaccine technologies. #Biotech #RareDiseases

💪 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

Soligenix raises $7.5M in public offering to advance rare disease pipeline, ensuring financial stability through 2026 and supporting critical clinical development programs #Biotech #RareDiseases

Scientific Director
CureGRIN Foundation

CureGRIN is looking for a brilliant, driven, junior PhD who will work remotely with researchers, clinicians, industry and patient families.

See the full job description on jobRxiv: https://jobrxiv.org/job/curegrin-foundation-27778-scientific-director/

#clinicaltrial #communityengagement #preclinical #rarediseases #Translationalmedicine #ScienceJobs #hiring #research
https://jobrxiv.org/job/curegrin-foundation-27778-scientific-director/?fsp_sid=3847

Scientific Director
CureGRIN Foundation

CureGRIN is looking for a brilliant, driven, junior PhD who will work remotely with researchers, clinicians, industry and patient families.

See the full job description on jobRxiv: https://jobrxiv.org/job/curegrin-foundation-27778-scientific-director/

#clinicaltrial #communityengagement #preclinical #rarediseases #Translationalmedicine #ScienceJobs #hiring #research
https://jobrxiv.org/job/curegrin-foundation-27778-scientific-director/?fsp_sid=3742

Tonix Pharmaceuticals receives FDA clearance for Phase 2 trial of TNX-2900, a promising intranasal treatment for Prader-Willi syndrome, advancing rare disease research and potential patient care #Biotech #RareDiseases

Soligenix receives FDA orphan drug designation for dusquetide, advancing rare disease treatment with promising phase 2a clinical trial results for Behçet's disease #RareDiseases #Biotech

Soligenix advances rare disease pipeline with innovative therapies, targeting transformative treatments for underserved medical conditions. Pivotal phase 3 cancer trial results expected in 2026. #Biotech #RareDiseases

Soligenix receives FDA Orphan Drug Designation for dusquetide in Behçet's Disease treatment, marking significant progress in rare disease therapeutics #Biotech #RareDiseases

🧬 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