@drtimos.bsky.social🚀 Big buzz at #ASGCT25: #GeneEditing & #CRISPR headlines, 20% data-driven talk, 85% positive vibes on breakthrough therapies for #DMD & #rarediseases!🧬 Download our full #socialmedia report! http://dlvr.it/TVlYyc #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT
@charityeos.bsky.socialMeet us at #RareSummit2026 on 7th October! Book your ticket 👉 https://www.camraredisease.org/raresummit26/
Bringing together experts in #RareDiseases, it's a valuable chance to learn more about #EosinophilicDiseases and the challenges holding back care.
@ipopi.bsky.socialPrimary and secondary immunodeficiencies may differ in origin, but their challenges can overlap. Our policy recommendations highlight five key areas for stronger EU action.
📄 Explore the full recommendations: bit.ly/4xgbJgq
@gmj-georgia.bsky.socialARPA-H is investing $160 million across seven research institutions to accelerate custom gene-editing therapies for rare genetic diseases. After admi… #ARPAH #Clinicaltranslation #Geneediting #Precisionmedicine #Rarediseases #ARPAH #Clinicaltranslation #Geneediting #Precisionmedicine #Raredisea…
@erdera.bsky.social🏆 Celebrate the people and initiatives making a difference in rare diseases.
Join the 15th #EURORDIS Black Pearl Awards on 24 Feb 2026, held in Brussels & online as part of #RareDiseaseDay celebrations.
🎥 Online participation is free.
👉 https://loom.ly/ClRoNdc
#RareDiseases #ERDERA
@erdera.bsky.social⏳ The deadline is approaching!
Want to expand your knowledge, connect with experts and help drive progress in rare diseases?
Apply for the Open Academy x ERDERA Schools 2027 before 16 October.
📍 Barcelona, 7-10 June 2027
🔗 Apply now: https://loom.ly/uZ0Iz8M
#ERDERA #RareDiseases
@hemehub.bsky.socialExplore the latest insights on Acute Intermittent Porphyria with Dr. Karl E. Anderson and Dr. Taha Bat in our upcoming live webinar!
⮕ Register: hemehub.org/live-webinars
⮕ Submit your cases: hemehub.org/case-submiss...
#AcuteIntermittentPorphyria #AIP #Porphyria #RareDiseases #Hematology
@2eubrussels.bsky.socialParliament’s SANT Committee wants an EU rare-diseases framework built around a major target: diagnosis within one year of the first medical consultation. It also seeks coordinated newborn screening and stronger European specialist networks. #RareDiseases #EUHealth
@ctgct.bsky.socialAt #CaringForRare2026, one message was clear: progress in rare diseases happens when researchers, clinicians and patients work together.
CTGCT's Gregor Cuzak joined discussions to advance gene & cell therapies.
Read more: ctgct.si/en/objave/ct...
@esendocrinology.bsky.socialDon't miss ESE Talks... #RareDiseases webinar - Artificial Intelligence in Rare Disease - with ESE, Endo-ERN and ESPE.
📅 6 October, 17:00-18:30 (CEST)
Join chairs Sofia Llahana and Elena Valassi as well as our expert speakers.
Register now! 👉https://ow.ly/L02w50Yrl6y
@drtimos.bsky.social🧬 Gene & Cell Therapy | #RareDiseases 🚀
#LucidQuest delivers sharp insights to help biotechs, investors & BD teams stay ahead in fast-moving markets.
📩 [email protected] or http://dlvr.it/TVhS2K
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights
@hlm4rare.bsky.socialJoin BRAINS FOR BRAIN FOUNDATION, #HLM4RARE and EUCOPE for "Unlocking European competitiveness: How the rare disease ecosystem drives health innovation."
📅 Wednesday, 30 September 2026 | 10:30-11:45 | Kursaal
Follow the session live 👉 www.hlm4rare.eu/european-hea...
@hlm4rare.bsky.social#HLM4Rare is built around four voices, each bringing a different kind of expertise and urgency to the table:
🔹 Patients and families
🔹 The 24 ERNs
🔹Industry
🔹 Policymakers
They're the ecosystem turning commitments into action for people living with #RareDiseases across Europe.
👉 hlm4rare.eu
@erdera.bsky.socialCan AI really deliver for rare diseases? 🤖 Join ERDERA at #WODCEurope 2026 for the pre-congress workshop "AI for Rare Diseases: From Promise to Practice". A lively debate on where AI can accelerate research and therapy development. 👉 Read more: https://loom.ly/FQvdeRY
#ERDERA #RareDiseases #AI
@stemcellnetwork.ca(2/2) pathways, or cellular systems in studying new or understudied genes associated with #RareDiseases. 🔬
Learn more about Dr. Flamier’s project and the SCN-RDMM Catalyst Grant program. ➡️ stemcellnetwork.ca/news/stem-ce...
@audubonballroom.bsky.socialComplicated released in 2025
"Children with a complex, mysterious illness and their parents are branded 'too complicated' and risk losing everything due to false accusations of medical child abuse."
(1/2)
#film #children #rarediseases #elvispresley #blackmusic #blacksky
@world-j-pediatr.bsky.socialComplex treatments save lives in rare pediatric diseases—but what happens next?
A new study highlights the vital need for long-term info & psychosocial care for families.
Open Access
link.springer.com/article/10.1...
@livinglfs.bsky.social
@4040rec.bsky.social