Orphanet’s Post

Today is #RareDiseaseDay! Despite promising research, rare endocrine conditions still have a major impact on the lives of millions of Europeans. Over 30 million people in Europe are affected by more than 400 endocrine rare diseases, along with numerous other severe rare diseases. Better diagnostics and improved treatment are needed now. European Reference Networks play a key role in ensuring all individuals with rare diseases get the care they need. Investing in a patient-centred approach is essential. Find out more: endo-ern.eu EUROPEAN ORGANISATION FOR RARE DISEASES EURORDIS #RareDiseaseDay #BecauseHormonesMatter

We will be presenting data from our 12-week #clinicaltrial evaluating #pemvidutide in subjects with #MASLD at The Liver Meeting® hosted by the American Association for the Study of Liver Diseases (AASLD) that support pemvidutide’s potential benefit on #MASH and its associated co-morbidities. Learn more: https://lnkd.in/ehYn4KNv.   #TLM24 #GLP1 #Glucagon 

Time is running out! ⏳    Act fast and submit your abstracts for the 20th ISDE World Congress for Esophageal Diseases #ISDE2024.   Don’t miss the deadline - submit your abstract by Wednesday, February 14, 2024 (11:59 pm EST) at https://lnkd.in/gs2aTm9d  #isde2024 #esophagus #esophageal #oesophagus #oesophageal 

Rare diseases concerns health conditons but transcend mere health considerations. In the realm of health for rare diseases we found: issues of equity, social justice, inequalities, and mental well-being. People living with rare diseases deal with aspects like fear, denial, isolation, overwhelming emotions and uncertainty. I take pride in my contribution to social equality across EU Member States through ERN-EuroBloodNet (European Reference Network on Rare Hematological Diseases). We are working towards a robust European Health Union by pooling expertise, implementing best practices, developing Continuous Medical Education courses, Patient Therpeutic Educational Program, Patients advocacy trainings, establishing registries, advancing epidemiology research strategies, and consultation for complex cases across European Union. Such a union is one that provides the means for cross-border health solutions in EU MS for individuals living with rare diseases. #RareDiseaseDay #ERNeu #HealthUnion #ShareCareCure

Translating Real world data (RWD) into real-world evidence (RWE) is critical to understand neuro-ophthalmological diseases. This review article proposes solutions to the key barriers that can limit translation of a collection of data into impactful clinical evidence. Read #OpenAccess https://lnkd.in/ezRaaYAk #Ophthalmology

📢 Only one week left to submit your abstract for the 20th ISDE World Congress for Esophageal Diseases #ISDE2024! 📝 Join us in making a significant impact on esophageal diseases through Poster, Oral, or Video presentations. Expand your network at the ISDE Congress and showcase your expertise to the professionals in the field! Check out the submission guidelines at https://lnkd.in/eGQRiPEg #Esophagus #EsophagealCancer #medicalresearch

The European Joint Programme on Rare Diseases announces the publication of a new article highlighting the past five years of the EJP RD.🌟 Titled "The European joint programme on rare diseases: building the rare diseases research ecosystem" this article published on the Rare Diseases and Orphan Drugs Journal delves into our collective achievements and ongoing efforts to foster a robust research environment for rare diseases.🚀 💡Read more about "The European joint programme on rare diseases: building the rare diseases research ecosystem" here: https://lnkd.in/dytdpMDQ #ERNeu #ERNs #HealthUnion #EU4Health #ShareCareCure European Health and Digital Executive Agency (HaDEA) European Commission

The Walden team is looking forward to collaborating with one of the world’s leading experts in Mendelian randomization to advance suPAR science and better understand the causative role of elevated suPAR levels in kidney diseases. Read the full release here: https://lnkd.in/ePRVaNDf #ChronicKidneyDisease #renaldisease #suPAR #MendelianRandomization #Walden #DrugDevelopment

Cooperation of all the Stakeholders in the Rare Diseases' field is required in order to build a patient-centric National Strategy for Rare Diseases. The #RareDiseaseInternationalConference2024 successfully highlighted the main areas of discussion. Access to innovation is one of the major issues. #RareDiseaseAwareness#OrphanDrugs#AccessToInnovation

Empathy knows no rarity. Let's champion equity and understanding for those with rare diseases. Rare Disease Day, observed annually on February 28 or 29 in leap years, is a global initiative fostering equity in social opportunities, healthcare, and diagnosis access for those with rare diseases. Established in 2008 by EURORDIS and 65+ patient organizations, it unites individuals, families, healthcare professionals, researchers, and the public in advocating for the 300 million people worldwide affected by rare diseases through various activities, including social media sharing and illuminating buildings. Every voice matters in the fight for rare diseases. Join us in raising awareness and making a difference! Organization for Rare Diseases India Rare Disease Research, LLC Rare Disease Research, LLC National Organization for Rare Disorders EURORDIS-Rare Diseases Europe Rare Disease Foundation #isupportrare #showyourstripes #raredisease #rarediseaseday #rarediseaseawareness #rarediseaseday2024 #healthcare #healthawareness #healthcareeducation