#rarediseases — Public Fediverse posts

Shane the Gamer: Belgian Gamer Michiel Vandeweert Dies at 28, Doubling Progeria Life Expectancy. “Belgian content creator and gamer Michiel Vandeweert has died at 28, more than twice as long as doctors expected when he was diagnosed as a child with progeria, the ultra-rare genetic disorder that causes rapid aging.”

Shane the Gamer: Belgian Gamer Michiel Vandeweert Dies at 28, Doubling Progeria Life Expectancy. “Belgian content creator and gamer Michiel Vandeweert has died at 28, more than twice as long as doctors expected when he was diagnosed as a child with progeria, the ultra-rare genetic disorder that causes rapid aging.”

This August, we're launching our #SummerOfScience series again! Stay tuned to hear about our most impactful stories from the past year.
First up is the launch of Screen4Care's tools to help diagnose #RareDiseases earlier and faster.
👶 A TREAT panel for testing newborns genetically for rare diseases using just a few drops of blood from a heel prick
📱 An app to reliably track a patients' symptoms as a diagnostic aide
👉 https://link.europa.eu/GYhXKX
#IHITransformingHealth #HorizonEU #HorizonEurope

This August, we're launching our #SummerOfScience series again! Stay tuned to hear about our most impactful stories from the past year.
First up is the launch of Screen4Care's tools to help diagnose #RareDiseases earlier and faster.
👶 A TREAT panel for testing newborns genetically for rare diseases using just a few drops of blood from a heel prick
📱 An app to reliably track a patients' symptoms as a diagnostic aide
👉 https://link.europa.eu/GYhXKX
#IHITransformingHealth #HorizonEU #HorizonEurope

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

Soligenix advances rare disease innovation using platform science to expand its therapeutic pipeline and address unmet medical needs affecting millions. #RareDiseases #Biotech

Soligenix advances rare disease innovation using platform science to expand its therapeutic pipeline and address unmet medical needs affecting millions. #RareDiseases #Biotech

Soligenix reports strong Phase 3 HyBryte data for cutaneous T-cell lymphoma, with response rates exceeding expectations. Interim analysis could accelerate commercialization path. #Biotech #RareDiseases

Soligenix reports strong Phase 3 HyBryte data for cutaneous T-cell lymphoma, with response rates exceeding expectations. Interim analysis could accelerate commercialization path. #Biotech #RareDiseases

Soligenix publishes peer-reviewed HyBryte clinical data demonstrating safety and efficacy for cutaneous T-cell lymphoma treatment. Phase 3 trial interim results expected Q2 2026. #Biotech #RareDiseases

Soligenix publishes peer-reviewed HyBryte clinical data demonstrating safety and efficacy for cutaneous T-cell lymphoma treatment. Phase 3 trial interim results expected Q2 2026. #Biotech #RareDiseases

Soligenix publishes HyBryte clinical data in peer-reviewed journal, highlighting safety and efficacy for cutaneous T-cell lymphoma treatment. Phase 3 interim analysis expected Q2 2026. #Biotech #RareDiseases

Soligenix publishes HyBryte clinical data in peer-reviewed journal, highlighting safety and efficacy for cutaneous T-cell lymphoma treatment. Phase 3 interim analysis expected Q2 2026. #Biotech #RareDiseases

Soligenix's SGX945 receives Promising Innovative Medicine designation from UK MHRA for Behçet's disease, advancing rare disease treatment development. #Biotech #RareDiseases

Soligenix's SGX945 receives Promising Innovative Medicine designation from UK MHRA for Behçet's disease, advancing rare disease treatment development. #Biotech #RareDiseases

Soligenix approaches pivotal 2026 with Phase 3 HyBryte trial for CTCL and SGX945 advancement for Behçet's disease. Multiple clinical catalysts expected. #Biotech #RareDiseases

Soligenix approaches pivotal 2026 with Phase 3 HyBryte trial for CTCL and SGX945 advancement for Behçet's disease. Multiple clinical catalysts expected. #Biotech #RareDiseases

Soligenix enters a pivotal 2026 with key clinical milestones ahead, including Phase 3 FLASH2 trial data for HyBryte in CTCL treatment and progress on SGX945 for Behçet's disease. #Biotech #RareDiseases

Soligenix enters a pivotal 2026 with key clinical milestones ahead, including Phase 3 FLASH2 trial data for HyBryte in CTCL treatment and progress on SGX945 for Behçet's disease. #Biotech #RareDiseases

Soligenix receives EU orphan drug designation for dusquetide in Behçet Disease, securing 10 years of market exclusivity. Phase 2a data demonstrated efficacy and safety. #Biotech #RareDiseases

Soligenix receives EU orphan drug designation for dusquetide in Behçet Disease, securing 10 years of market exclusivity. Phase 2a data demonstrated efficacy and safety. #Biotech #RareDiseases

Soligenix receives EU orphan drug designation for dusquetide in Behcet Disease, securing 10 years of market exclusivity. Builds on Phase 2a efficacy data. #Biotech #RareDiseases

Soligenix receives EU orphan drug designation for dusquetide in Behcet Disease, securing 10 years of market exclusivity. Builds on Phase 2a efficacy data. #Biotech #RareDiseases

Soligenix advances Phase 3 FLASH2 trial for cutaneous T-cell lymphoma with interim analysis expected Q2 2026. Company also secured orphan drug designation for dusquetide in Behçet's Disease. #Biotech #RareDiseases

Soligenix advances Phase 3 FLASH2 trial for cutaneous T-cell lymphoma with interim analysis expected Q2 2026. Company also secured orphan drug designation for dusquetide in Behçet's Disease. #Biotech #RareDiseases

Soligenix's SGX945 earns Promising Innovative Medicine designation in the UK, strengthening its rare disease pipeline. The designation builds on FDA orphan drug recognition for dusquetide. #Biotech #RareDiseases

Soligenix's SGX945 earns Promising Innovative Medicine designation in the UK, strengthening its rare disease pipeline. The designation builds on FDA orphan drug recognition for dusquetide. #Biotech #RareDiseases

Soligenix advances HyBryte clinical credibility with peer-reviewed publication in Expert Opinion on Investigational Drugs, strengthening validation for its cutaneous T-cell lymphoma therapy. #Biotech #RareDiseases

Soligenix advances HyBryte clinical credibility with peer-reviewed publication in Expert Opinion on Investigational Drugs, strengthening validation for its cutaneous T-cell lymphoma therapy. #Biotech #RareDiseases

Soligenix advances Phase 3 CTCL trial with interim analysis expected Q2 2026. Company secures orphan drug designation for dusquetide in Behcet's Disease, strengthening rare disease pipeline. #RareDiseases #Biotech

Soligenix advances Phase 3 CTCL trial with interim analysis expected Q2 2026. Company secures orphan drug designation for dusquetide in Behcet's Disease, strengthening rare disease pipeline. #RareDiseases #Biotech

Soligenix receives European Commission orphan drug designation for SGX945 in Behçet's disease treatment, validating its rare disease pipeline strategy. #Biotech #RareDiseases

Soligenix receives European Commission orphan drug designation for SGX945 in Behçet's disease treatment, validating its rare disease pipeline strategy. #Biotech #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 reaches 50-patient milestone in Phase 3 HyBryte trial, showing 48% response rate for cutaneous T-cell lymphoma treatment. Promising results signal potential breakthrough in rare disease therapy. #Oncology #RareDiseases

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

📊 Global Voxzogo Market Outlook 2026–2030

Growth driven by rising rare disease investments, genetic research, and demand for disease-modifying therapies.

Key trends:
• Targeted genetic therapies
• Early intervention focus
• Personalized pediatric treatments

North America leads the market.

📥 Get free sample:
https://www.thebusinessresearchcompany.com/report/voxzogo-global-market-report

#Healthcare #Pharma #RareDiseases #MarketResearch

📊 Vpriv Market Outlook to 2030

Growth driven by rising healthcare spending, rare disease treatment access, and advancements in enzyme therapies.

Key trends:
• Personalized dosing strategies
• Recombinant enzyme tech
• Long-term disease management

North America leads the market.

📥 Get free sample:
https://www.thebusinessresearchcompany.com/report/vpriv-global-market-report

#Healthcare #Pharma #RareDiseases #MarketResearch

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/

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

PNH & aHUS Market Growth Report 2026–2035 📊

The Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS) market is projected to grow from $5.97B in 2025 to $6.41B in 2026, driven by rising rare disease diagnosis, advanced testing, and next-generation complement inhibitors.

Get the free sample report:
https://www.thebusinessresearchcompany.com/sample.aspx?id=24357&type=smp

#Healthcare #RareDiseases #Biotech #MarketResearch

💡 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

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