explore | Grilled Cheese

A 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/2026-08-28/21167

A 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/2026-08-28/21169

Don'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.

👶 September is #NewbornScreening Awareness Month. Across Europe, access to comprehensive newborn screening remains uneven, contributing to avoidable inequalities in diagnosis and care.
Learn more in ERDERA's latest #ScientificPill👉 https://loom.ly/28UA3H8
#RareDiseases #ERDERA #HealthEquity

🚀 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/TVSzLc #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT

A huge thank you to all speakers, and especially to the patient organisations who made Session 8 on rare diseases & (gene) therapies so powerful. Your voices matter and the lively discussion showed why patients must be at the heart of research. 💙 #RareDiseases

Yesterday I had the honor of being recognized for my work in genetics & genomics of human diseases with the "Matilde Montoya Award for Women in Biological Sciences and Medicine". I am grateful for this honor & motivated to continue pushing genomics to improve the diagnosis of #RareDiseases

From genotype to phenotype 🧬 Thursday’s final session connected variant biology, precision medicine and therapy. Stanley T. Crooke presented n-Lorem’s approach to developing personalized ASOs for patients with pathogenic mutations—free for life. #CCPConference2026 #RareDiseases

From models to therapies 🔬
Yesterday’s Session 2 at #CCPConference2026 showcased how advanced preclinical models can bridge disease mechanisms and treatment:
The key message: the right model can turn genetic insight into therapeutic opportunity.
#RareDiseases #Phenogenomics

🧬 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/TVQJZZ
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights

📬 Miss the September newsletter? Catch up on the latest updates and highlights from across ERDERA and the rare disease research community. Read it here 👉 https://loom.ly/VxVn5mI
📩 Don’t miss the next issue, subscribe now: https://loom.ly/TFRXEBY
#ERDERA #RareDiseases

🌍 How can Europe better address the needs of people living with primary and secondary immunodeficiencies?

🔗 Read the full recommendations: bit.ly/4xgbJgq

#IPOPI #Immunodeficiencies #PrimaryImmunodeficiencies #EUHealth #HealthPolicy #RareDiseases

🎉First RealiseD publication available!
The assessment of randomisation methods used in clinical trials to minimise bias.

📰Read it: https://loom.ly/iSuP3KE
🔓Access article: https://doi.org/10.1371/journal.pone.0339427

#RareDiseases @ihieurope.bsky.social

Sam Barrell, chief executive of LifeArc, explains what we need to do to turn discovery science into patient benefit and why the UK has the potential to position itself as a market leader for rare diseases.

healthcaretoday.com/article/life...

#RareDiseases #LifeSciences #ClinicalTrials

“People with #ChronicPain don’t get a break from feeling tired. We just have to keep going… The pain can be so intense that it just exhausts…physically and mentally.” When Your Chronic Pain Leaves You in a Constant State of Tiredness buff.ly/2QDK9qf #PainAwarenessMonth #RareDiseases

🚀 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/TVL5wC #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT