(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...
@stemcellnetwork.ca
@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...
@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/TVcPqP #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT
@sma-europe.bsky.socialKind reminder! The registration and the call for abstracts still open for the 2nd International Clinical Care Symposium on SMA, taking place on 4–5 March 2027 in Prague, Czech Republic. 📣
🔗https://www.sma-europe.eu/clinical-care-symposium-prague-2027
@4040rec.bsky.social
@jmirlatestresearch.bsky.socialApplication of aiAtlas Version 1.2 to Simulate Variant Gene Function Restoration and Rescue Thresholds in Rare Diseases: Validation Case Study #ArtificialIntelligence #Genomics #RareDiseases #GeneTherapy #BiomedicalResearch
@dhsc-answers.bsky.socialA question on #RareDiseases #AlliedHealthProfessions tabled by Lord Kamall on 09-09-2026 has been answered by Baroness Merron. https://questions-statements.parliament.uk/written-questions/detail/2026-09-09/HL3331
@dhsc-answers.bsky.socialA question on #RareDiseases #MedicalTreatments tabled by Mr Lee Dillon on 11-09-2026 has been answered by Mr James Frith. https://questions-statements.parliament.uk/written-questions/detail/2026-09-11/29295
@dhsc-answers.bsky.socialA question on #RareDiseases #HealthServices tabled by Dr Luke Evans on 02-09-2026 has been answered by Mr James Frith. https://questions-statements.parliament.uk/written-questions/detail/2026-09-02/26638
@eurordis.bsky.social👶 Where a child is born in Europe can shape their chances of being screened for a rare disease.
In a new Mediaplanet article, EURORDIS’ Gulcin Gumus explores why more equitable newborn screening across the EU matters.
@hemehub.bsky.social🩸 Join us for an upcoming live webinar with Karl E. Anderson, MD and Taha Bat, MD on Acute Intermittent Porphyria, featuring case-based discussions and real-world clinical perspectives.
⮕ Register: hemehub.org/live-webinars
#AcuteIntermittentPorphyria #AIP #Porphyria #RareDiseases #Hematology
@dhsc-answers.bsky.socialA question on #RareDiseases #Drugs tabled by Mr Luke Charters on 11-09-2026 has been answered by Mr James Frith. https://questions-statements.parliament.uk/written-questions/detail/2026-09-11/29268
@realised-ihi.bsky.social🔍 Did you know that six European Reference Networks (ERNs) are helping shape the future of rare disease clinical trials through RealiseD?
🔍 Learn more about ERNs and their role in the project: https://loom.ly/8uvU3Ms
#RealiseD #RareDiseases #ERNs #ClinicalTrials #HealthInnovation
@gmj-georgia.bsky.socialBreaking: Talos, a new automated tool, is reshaping rare disease diagnostics by systematically reanalysing stored genomic d… #Artificialintelligence #Clinicalgenomics #Genomicmedicine #Precisiondiagnostics #Rarediseases #Artificialintelligence #Clinicalgenomics #Genomicmedicine #Precisiondiagno…
@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/TVYYdX
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights
@nordrare.bsky.socialNORD applauds Reps. Bilirakis, Barragán, Cammack, Auchincloss, Kean and Mullin for introducing the Priority for Pediatric Cures Act to make the Rare #Pediatric Disease #PRV Program permanent.
On behalf of children with #RareDiseases, NORD strongly urges Congress to pass this bipartisan legislation.
@eurordis.bsky.social📣 Kickstart Your Advocacy starts on 24 September!
Join Nicole Faccio & Julien Poulain for a FREE webinar on using storytelling to strengthen your advocacy and make your voice heard.
👥 For young people under 30
👉 Register: go.eurordis.org/advocacy-web...
@killbait-uk.bsky.socialA Mother's Struggle and Hope for Her Daughter with Complex Medical Needs
🤖 IA: It's not clickbait ✅
👥 Users: It's not clickbait ✅
👇👇👇
@dhsc-answers.bsky.socialA question on #Drugs #RareDiseases tabled by Peter Dowd on 28-08-2026 has been answered by Mr James Frith. https://questions-statements.parliament.uk/written-questions/detail/2026-08-28/21168