Join the World Orphan Drug Congress USA 2026! Connect with leaders, regulators, and patient advocates to advance rare disease therapies, clinical trials, and patient access strategies from June 9–11 in Boston.
For more info: buff.ly/qjKvjPO
#MedSky #OrphanDrugs #RareDisease #PatientAdvocacy
New paper w/Paul Martin & Jin Ding: “Why is the pharmaceutical industry investing in targeted therapies? The emergence of “premium pharma" | How #orphandrugs #ATMPs #TargetedTherapies reshape #BioPharmaInnovation #HTA sciencedirect.com/science/article/pii/S0277953625012195 @sheffielduni.bsky.social
5 rare disease breakthrough drugs approved in China.
Faster approvals are turning “untreatable” into treatable.
When treatment becomes possible,
rare no longer means hopeless.
📩 info@dengyuemed.com
🔗 dengyuemed.com
#RareDisease #OrphanDrugs #ChinaPharma #Biotech #DrugApproval
American Trypanosomiasis (#ChagasDisease) could be treated using newly developed #Benznidazole (BNZ) orally disintegrating tablets.
Check out the full study in #JPP:
🔗 doi.org/10.1093/jpp/...
#OrphanDrugs
Increased focus on rare genetic disorders is shaping this niche market. 🧬🩺 www.marketresearchfuture.com/reports/broo...
#OrphanDrugs
Canavan Disease Treatment Market advances with ongoing gene therapy and orphan drug development. www.marketresearchfuture.com/reports/cana... #OrphanDrugs
General 340B expertise ≠ orphan drug expertise.
The FDA OOPD database, manufacturer discretionary pricing, biosimilar analysis—it's specialized work.
11+ years in this niche. $200K+ avg client savings.
#340B #OrphanDrugs #Healthcare
Blueprint graphic with the number 1 and text: “Plan Early, Plan Smart.”
Blueprint graphic with the number 2 and text: “Build on Solid Strategy.”
Blueprint graphic with the number 3 and text: “Validate and Align Early.”
Blueprint graphic with the number 4 and text: “Be Ready—From Dossier to Decision,” plus a small image of the JCA report.
ow.ly/y4I550XuUw1 EU Joint Clinical Assessments are reshaping orphan drug access in Europe.
Download RTI Health Solutions’ free report for the key challenges, evidence needs, and strategies to stay ahead.
#OrphanDrugs #RareDisease #JCA
3e
Repurposing medications to treat people with rare diseases.
https://ow.ly/ceYk50XuG2b
#RareDiseases #MedicationRepurposing #HealthInnovation #PharmaceuticalResearch #OrphanDrugs
At #WODC, Ralf-Dieter Hilgers, RealiseD Coordinator from Sigmund Freud Private University, presented the paradigm shift RealiseD is leading in #UltraRareDisease research. 💡This will take shape in Playbooks—practical guides for better, fairer clinical trials. #RareDisease #RealiseD #OrphanDrugs
That’s a wrap on an inspiring #WODC2025! 🌍
The #ERDERA team joined panels, sparked valuable exchanges, and connected with many eager to collaborate and contribute to advancing #RareDisease research in Europe. 💬 Let’s keep the momentum going! 💪 #OrphanDrugs #WODC
Hello #WODC2025 👋
Day 1 kicks off with workshops—and we’re thrilled to be part of it!
🚀 ERDERA, RealiseD & Inventis is leading the biggest session on innovative methodological approaches. Stay tuned for session insights here & on our website!
#RareDiseases #OrphanDrugs #ERDERA
🎉 Hello #WODC2025! RealiseD, ERDERA & Inventis kicked things off with the congress’s largest workshop on accelerating rare disease drug development.
📢 Workshop insights coming soon on our website—stay tuned!
#RareDiseases #OrphanDrugs #RealiseD
🎉 One week to go for the World Orphan Drug Congress 2025 — a global forum driving innovation and collaboration in #OrphanDrugs and #RareDiseases. 📍 Visit ERDERA at Booth 10.512!
🎟️ Free tickets for patient groups: https://loom.ly/vDD9hwI
More info: https://loom.ly/a5bnWok
In rare diseases, the “n of 1” matters, and efficacy narratives help tell that story with clarity and impact. For more on #EfficacyNarratives in #RareDiseases, read our latest LinkedIn post: www.linkedin.com/feed/update/...
#ClinicalDevelopment #RegulatoryStrategy #OrphanDrugs
Welchen Nutzen haben #OrphanDrugs wirklich? Wie misst man ihn – und was darf das kosten? Teil 2 des Interviews mit zwei #Experten des Pharmaunternehmens #Astellas.
pharma-fakten.de/news/orphan-...
⏳ Less than a month to #WODCEurope!
On 27 Oct at 10:00, ERDERA, RealiseD & INVENTS lead a workshop on innovative trial design & evidence generation in #RareDiseases, chaired by Daria Julkowska.
Don’t miss it! Register with code ERDERA20 🔗 https://loom.ly/kg5KUrA
#OrphanDrugs #ClinicalResearch
FYI: LucidQuest Views >>> Rare Diseases Weekly News – August 12th 2025 #News #OrphanDrugs #MedicalBreakthroughs #Raxone Comment below!
ICYMI: LucidQuest Views >>> Rare Diseases Weekly News – August 12th 2025 #News #OrphanDrugs #MedicalBreakthroughs #Raxone Comment below!
LucidQuest Views >>> Rare Diseases Weekly News – August 12th 2025 #News #OrphanDrugs #MedicalBreakthroughs #Raxone Comment below!
🎯 Working on #RareDisease or #DrugRepurposing? Join the #WODC2025 in Amsterdam, 28–29 Oct. EATRIS is a partner of the event — submit a poster & be part of the #PatientCentred innovation wave.
👉 Use our 20% discount code: eatris.eu/events/world-orphan-drug... #OrphanDrugs
LUNGevity Foundation - What Are Orphan Drugs, and How Can They Be Leveraged for SCLC?
@lungevity.bsky.social
oncodaily.com/blog/orphan-...
#OncoDaily #Oncology #Cancer #Health #Medicine #MedEd #MedOnc #MedNews #LUNGevity #SCLC #LungCancer #OrphanDrugs
🔵 One’s already at the FDA’s desk.
👉 The rest are pacing fast—each one targeting <10K U.S. patients.
Brittle bones. Rare bile damage. Zero approved options.
♻️ Repost 💡 Follow @sbokhiria.bsky.social
#RareDisease #Biotech #OrphanDrugs #DrugPipeline #ClinicalTrials
Fighting fatigue in rare form! Targeted therapies are transforming myasthenia gravis treatment.
#MyastheniaGravis #RareDiseases #NeuroCare #Immunotherapy #OrphanDrugs
www.marketresearchfuture.com/reports/myas...
Battling rare blisters! Therapies for pemphigus vulgaris are gaining traction in immuno-dermatology.
#PemphigusVulgaris #RareDiseases #DermatologyCare #AutoimmuneTreatment #OrphanDrugs
www.marketresearchfuture.com/reports/pemp...
FYI: LucidQuest Views >>> Rare Diseases Weekly News – June 19th 2025 #News #BeckerMuscularDystrophy #Duchenne #OrphanDrugs Comment below!
🚨 New FDA Draft Guidance: Rare Disease Drug Development
FDA outlines the critical role of pre-IND meetings, regulatory flexibility, and early planning for orphan drug programs.
💡 Read more: compliancearchitects.com/ra...
#FDA #RareDisease #OrphanDrugs #DrugDevelopment #ComplianceArchitects
ICYMI: LucidQuest Views >>> Rare Diseases Weekly News – June 19th 2025 #News #BeckerMuscularDystrophy #Duchenne #OrphanDrugs Comment below!
LucidQuest Views >>> Rare Diseases Weekly News – June 19th 2025 #News #BeckerMuscularDystrophy #Duchenne #OrphanDrugs Comment below!
