#Rarediseases📄
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erdera.bsky.social
⭐ New from #IRDiRC! The Funding Models Toolbox supports patient orgs in navigating rare disease research funding.
📎 Toolbox: loom.ly/P67cbCg
📄 Publication: loom.ly/Q7oHLEk
ℹ️ Task Force info: loom.ly/bj02hvQ
#RareDiseases #PatientAdvocacy #Research
May 20, 2025 at 8:16 AM
varechavala.bsky.social
🚨 We're hiring! 🚨

Join us at Biobizkaia 🌍 as part of the MSCA-DN EffecT project! We’re offering a fully funded #PhD position UPV/EHU. 🎓

📌 Focus on:
🔬 #Antisense #oligonucleotides
🧬 #Splicing
🌟 #Rarediseases📄

Apply now: euraxess.ec.europa.eu/jobs/296420
Info here: www.effect-dn.eu/candidates
12 PhDs on Antisense oligonucleotide research to foster the full therapeutic potential of antisense technology across tissues
EFFecT (European Training Program to Foster the Full Therapeutic Potential of Antisense Technology across Tissues) is a Marie Skłodowska-Curie Doctoral Network (2025-2028), offering 12 PhD positions.
euraxess.ec.europa.eu
January 14, 2025 at 10:05 AM
scienceopen.bsky.social
📄 'Exploring a Mechanism-Based Therapeutic Approach for #ZC4H2 Haploinsufficiency' - a DrugRxiv #Preprint on #ScienceOpen:

#️⃣ @repo4eu.bsky.social #DrugRepurposing #RareDiseases #PrecisionMedicine #Neurodevelopment
Exploring a Mechanism-Based Therapeutic Approach for ZC4H2 Haploinsufficiency
<p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="first" dir="auto" id="d350638e185">This report explores a therapeutic hypothesis for addressing the potential effects of a l...
drugrepocentral.scienceopen.com
August 22, 2025 at 2:32 PM
scienceopen.bsky.social
📄 'Strategies to Advance Drug Repurposing for Rare Diseases' - a DrugRxiv #Preprint on #ScienceOpen:

#️⃣ @repo4eu.bsky.social #DrugRepurposing #RareDiseases #NonProfitResearch
Strategies to Advance Drug Repurposing for Rare Diseases
<p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="first" dir="auto" id="d1369019e93">While the number of novel treatments for rare diseases has grown substantially over the p...
drugrepocentral.scienceopen.com
August 22, 2025 at 2:29 PM
synthemaeu.bsky.social
How is the EU addressing the challenges of Sickle Cell Disease? This paper co-authored by #SYNTHEMA partners explores the role of ERN-EuroBloodNet in advancing care, collaboration & research for rare haematological diseases.

📄 Read more: bit.ly/44CHlSP

#RareDiseases #SickleCell #Hematology
April 22, 2025 at 8:32 AM