RARE DISEASES INTERNATIONAL

🎉 Great news: We are thrilled to share that our CEO Alexandra Heumber Perry has been selected as the winner of the Award for Outstanding Achievements in Rare Diseases for the category of International organizations to recognize her great efforts and achievements at RARE DISEASES INTERNATIONAL by the MENA Organization for Rare Diseases during the Opening Ceremony of the MENA Congress for Rare Diseases in Abu Dhabi, UAE. Thank you to Dr. Ayman El-Hattab This recognition celebrates the incredible efforts and dedication of the entire RDI network and the global rare disease community. It highlights our shared commitment to equity, inclusion, and action for the 300 million persons living with a rare disease worldwide. 🗣️ Alexandra Heumber Perry shared these inspiring words on receiving the award: “I’m truly honored and humbled to receive the Outstanding Achievement Award in Rare Diseases on behalf of Rare Diseases International. This recognition means so much to the entire global rare disease community. It celebrates the passion, resilience, and determination of everyone working to ensure no one is left behind because their disease is rare. It comes at a pivotal moment, as momentum builds toward the adoption of the first-ever WHA Resolution on Rare Diseases. With over 250 organizations united in our global coalition, we are ready to step forward together and turn this milestone into meaningful, lasting change for the 300 million people living with a rare disease worldwide, as we are rare but together we are many, united and everywhere.” 🌐 Thank you to everyone who continues to stand with us—this award belongs to all of you. Remember: You matter. Your health matters. Your voice matters. #RareDiseases #GlobalHealth #RDI #EquityInHealth #LeaveNoOneBehind #WHAResolution #MENARareDiseases #Resolution4Rare #PLWRD #Health4All #UHC MENA Organization for Rare Diseases

Today is #WorldHemophiliaDay: a day dedicated to celebrating the Hemophilia community around the world! Together with RDI member World Federation of Hemophilia / Fédération mondiale de l'hémophilie, we honor people around the world who live with hemophilia. This year’s theme is “Access for all: Women and girls bleed too”. Today, women and girls with bleeding disorders (WGBDs) are still underdiagnosed and underserved. The global health community has the power—and the responsibility—to change this. Through recognition, diagnosis, treatment, and care, the quality of life of women and girls will improve, and the bleeding disorders community will become stronger. We stand with WFH in calling for improved access to diagnosis, treatment and care for the hemophilia community and for everyone living with a rare disease. 🩸 Learn more about hemophilia: https://lnkd.in/eG4VKVAj 🌐 Learn about how we can improve the lives of people living with hemophilia and other rare diseases through the #Resolution4Rare at the #WHA78: https://lnkd.in/dPkjSDJk #WHD25

💊 Last week, RDI hosted a public webinar exploring how the WHO Essential Medicines List can be leveraged to improve access to rare disease treatments. During the session, RDI shared key insights from its Essential Medicines Working Group, offering practical guidance on EML applications and shedding light on the barriers faced by advocates working to include rare disease treatments.   🗣️ A dynamic panel of global experts emphasized the power of a collaborative, multistakeholder approach to advancing care for the 300 million people living with a rare disease. 📢 The webinar also marked the launch of RDI’s Call to Action, advocating for stronger inclusion of rare diseases in national and global essential medicines policies.  🔗 If you missed the webinar, watch the full recording here: https://lnkd.in/dNsijDTw 🇪🇸 Versión con traducción al español (comienza à las 03:00): https://lnkd.in/eqEDmEzh 💡 Stay tuned: RDI will soon publish a full report detailing the outcomes of the Working Group and the potential of the EML to drive meaningful change. #RareDiseases #EssentialMedicines #AccessToMedicines #WHO #UHC #CallToAction #GlobalHealth #HealthEquity #RDI

RDI is hiring! Are you looking for an opportunity to improve the lives of Persons Living with a Rare Disease (#PLWRD) and their families? We are hiring a Senior Manager of Global Programmes to lead the development and implementation of programmes for PLWRD focusing on access to timely diagnosis care and treatment, and improved quality of life for PLWRD and their families, as well as strengthening a global network. Fully remote position based in Italy, France or Belgium. Find out more: https://lnkd.in/d5V9X7aD #Hiring #ScienceJobs #RareDiseaseJobs #PublicHealthJobs

Why support the #Resolution4Rare? Because we are all connected to someone who lives with a rare disease – whether a family member, friend, neighbor or coworker. Yet, for too long, rare diseases have been overlooked in global health policies, leaving over 300 million people worldwide without adequate support, diagnosis, or treatment. That’s why the World Health Assembly (WHA) Resolution on Rare Diseases is so critical. By recognizing rare diseases as a global health priority, the #Resolution4Rare paves the way for stronger healthcare systems, increased research, and better access to life-saving treatments. It is a call for equity—ensuring that no one is left behind simply because their condition is considered rare. Now is the time to act. Support the rare disease community by urging policymakers, healthcare leaders, and global institutions to support this resolution and commit to tangible actions that improve the lives of those affected. Together, we can drive change and create a future where people with rare diseases receive the care and recognition they need and deserve. What about you? Why will you support the #Resolution4Rare? Find out more: https://lnkd.in/dPkjSDJk #UHC #HealthForAll #rarediseases #WHA

🚨 Happening today! Don’t miss our public webinar on leveraging the WHO Essential Medicines List (EML) for rare disease treatments! 💊 🕑 10 April | 14:00 CEST 🔗 Register: https://lnkd.in/d6XBsDeq The WHO EML is a vital tool for advancing Universal Health Coverage (UHC)—but its potential to improve access to rare disease medicines is still underrecognized. Join us to: ✅ Understand the EML’s role in rare disease access. ✅ Explore findings from RDI’s Essential Medicines Working Group. ✅ Hear from global experts and advocates. ✅ Support our Call-to-Action for greater inclusion of rare diseases in essential medicines policies. 🗣️ Speakers include: Dr. Chiuhui Mary Wang, Chiara Tovani, Roberta Anido de Pena, Roberta Luzzi, Salome Mekhuzla, Prof. Sangeeta Sharma, Prof. GILLES VASSAL. 🌎 Spanish interpretation available #RareDiseases #UHC #WHO #EssentialMedicines #HealthForAll #PublicWebinar #GlobalHealth #AccessToMedicines

RARE DISEASE EXPERTISE, CENTERS & NETWORKS: Results of the Public Survey 🔬 🌍 How is rare disease expertise organized worldwide? Our latest survey gathered insights from 228 respondents across 63 countries, revealing how experts collaborate, where centers for rare diseases exist, and how cross-border clinical services operate. 💡 The findings helped identify existing expertise and care-providing centers, laying the foundation for stronger collaboration. Connecting experts and centers for rare disease is key to improve diagnosis, care and treatment for people living with rare diseases globally. 📱 If you'd like to learn more, check out the full report here: https://lnkd.in/dCpCS9_Y  #RareDiseases #PublicSurvey #Report #PublicHealth #CrossBorderServices #Diagnosis

Today is #WorldHealthDay, and this year’s theme "Healthy Beginnings, Hopeful Futures" reminds us that investing in maternal and newborn health isn’t a cost—it’s a commitment to a healthier, more equitable world🤰   🌍 At Rare Diseases International, we recognize that early health interventions, access to diagnostics, and inclusive care from birth lay the foundation for lifelong well-being. We work globally to ensure that rare diseases are integrated into universal health coverage—starting with maternal and newborn care as essential entry points.   🔍 Over 70% of rare diseases begin in childhood, and many are life-threatening without timely diagnosis and treatment. Tools like newborn screening and access to lifesaving therapies must be available from the very beginning of life to ensure no child is left behind.   👶 Our vision is a world where no one living with a rare disease is left behind, beginning with equitable care for mothers, babies, and children everywhere.   #HealthForAll #RareDiseases #MaternalHealth #NewbornScreening #NewbornCare #ChildHealth #GlobalHealth #EquityInHealth #UHC #WHOday World Health Organization

Why support the #Resolution4Rare? Because rare conditions are not as rare as you might think. There are over 7,000 identified rare conditions, and 300 million persons living with a rare disease (PLWRD) around the world. While PLWRD are collectively not rare, they face unique challenges in accessing diagnosis, treatment and care. That is why rare diseases must be a global health priority. By supporting the #WHA Resolution on Rare Diseases, you can help improve outcomes for #PLWRD wherever they live. How can you show your support? 📣 Share this post 📖 Read our call to action: https://lnkd.in/dcPx4xZQ 🖊️ Join our coalition in support of the WHA Resolution on Rare Diseases: https://lnkd.in/d2k6JsP2 🔍 Learn more by visiting our website: https://lnkd.in/dPkjSDJk

🚨 Just 1 week to go! Join our public webinar on the WHO Essential Medicines List (EML)! https://lnkd.in/d6XBsDeq 💊 What is the EML? EML is a critical instrument for advancing Universal Health Coverage (UHC) by ensuring identification of essential medicines. However, its role in rare disease access remains underrecognized. This webinar will explore how the EML can serve as a powerful tool to improve access to treatments for persons living with a rare disease (PLWRD) at both global and national levels. 💡 Why attend? • Understand the WHO EML and its significance for rare diseases. • Discover key findings from RDI’s Essential Medicines Working Group. • Learn about our Call-to-Action to leverage EML for RD medicines. 🗣️ Hear from leading experts: • Dr Chiuhui Mary Wang – Global Programme, Rare Diseases International • Chiara Tovani – Global Programme, Rare Diseases International • Roberta Anido de Pena Anido – Secretaria de Comisión Directiva, Federación Argentina de Enfermedades Poco Frecuentes - FADEPOF • Roberta Luzzi – Senior Director, Global Medical Affairs HIDO, Chiesi Group • Salome Mekhuzla – Director, Global Development, World Federation of Hemophilia / Fédération mondiale de l'hémophilie (WFH) • Prof. Sangeeta Sharma – Professor, Neuropsychopharmacology, IHBAS & President (Hon), DSPRUD • Prof. GILLES VASSAL – Professor, Gustave Roussy Cancer Center, SIOP & SIOP Europe 🌎 Spanish interpretation will be available during the webinar. 🔗 Register now! https://lnkd.in/d6XBsDeq  #RareDiseases #UHC #WHO #EssentialMedicines #PublicWebinar