Johan Prevot

DAY 2 – The future of medicine took center stage at King Chulalongkorn Memorial Hospital for the @ipopi_info | SEAPID | @prmdcu | @ErasmusMC Meeting in #Bangkok. 🔬 Focus areas included: • CAR-T cell therapies • RAG1 / RAG2 gene therapy • Prime editing as a candidate therapy for IEI • “Intriguing Cases” from across Southeast Asia We were there — ensuring the patient voice remains central as innovation accelerates from bench to bedside. Bolder therapies. Deeper collaboration. Brighter futures for people living with IEI. #IPOPI #IEI #GeneTherapy #CART #Immunology #PatientVoice

🎬 “The Life Journey with Primary Immunodeficiencies” Our new mini documentary shares the powerful life journeys of people living with PIDs. It highlights the reality of delayed diagnosis, the challenges of accessing treatment, and what it truly means to live every day with a chronic condition. 🎥 Watch it now and help us amplify these voices: youtu.be/_0mRituivcc This video was supported by an unrestricted grant from #Takeda. #PIDAwareness #RareDiseases #PatientVoices

A critical time in the EU - 4 countries collect about half of all EU plasma by engaging both public AND private sectors. Others only rely on public sector plasma collection. Patients need more plasma. The solution is obvious — so why isn't everyone on board?

📍 IPOPI at the European Medicines Agency (EMA) IPOPI is taking part in the PCWP–HCPWP Joint Meeting at the @EMA_News in Amsterdam, represented by @OtiliaStanga. Key discussions include: • HTA Regulation • Medicine shortages • Union list of critical medicines • New pharmaceutical legislation • Patient experience in evidence generation Ensuring the patient voice is reflected in EU regulatory and policy discussions remains a priority for #IPOPI. #PatientVoice #EMA #RareDiseases #HealthPolicy #MedicinesAccess

🎬 Sharing real stories to raise awareness about Pulmonary Alveolar Proteinosis (#PAP) By speaking openly about their journeys, challenges, and resilience, they help shed light on a rare and often misunderstood disease — and contribute to greater awareness, understanding, and dialogue within the community. We are deeply grateful to Eduardo and Sara for their openness, trust, and willingness to share their #stories. 👉 Watch the mini-documentary and hear their voices: youtube.com/watch?v=mSPXlm… #RareDiseaseAwareness #PatientAdvocacy #MiniDocumentary #HealthCommunication

A milestone moment for rare disease communities across Europe! ✨ Today at the High-Level Meeting, @ipopi_info proudly signed the EU Declaration on #RareDiseases, joining partners and policymakers in a united call to improve the lives of millions of people living with rare conditions. By signing this Declaration, IPOPI reinforces its commitment to: 🔹 Advance equitable access to #diagnosis, treatment and care 🔹 Strengthen early #detection and newborn screening 🔹 Support innovation in #therapies for rare and complex conditions 🔹 Ensure patient voices remain at the heart of EU #policy This is a decisive step towards a stronger, coordinated European approach — one that can truly transform outcomes for patients and families. Together, we move closer to a future where no rare disease patient is left behind.

📝 Our latest educational leaflet is now available! A tool to connect patients, families, and healthcare professionals and foster a stronger PID community. Get it here: ipopi.org/publications/l… #IPOPI #SecondaryImmunodeficiency #Leaflet

🚨 Pulmonary Alveolar Proteinosis (PAP) can silently affect the lungs of people living with PIDs — and often goes undiagnosed. 🎥 Our new mini-documentary brings expert insights and patient voices to light a hidden but life-threatening challenge. 🔗 youtube.com/watch?v=mSPXlm… #RareDiseases #PIDs #PAP #DiagnosticDelay #PAPdoc

🧬 New hope for people living with MUNC13-4 deficiency For individuals affected by MUNC13-4 deficiency, new treatment options are urgently needed. A new Phase I/II clinical trial is now open, exploring an innovative gene therapy approach designed to correct the underlying cause of the disease. 📍 The clinical trial is currently available at @hopital_necker in Paris. 🌍 Patient recruitment is open across Europe. 👉 Learn more about the trial: euclinicaltrials.eu/search-for-cli… 💬 If you or someone you care for is affected by MUNC13-4 deficiency, we encourage you to speak with your specialist to understand whether this trial may be relevant. #GeneTherapy #MUNC134 #IPOPI

✨Season’s Greetings from IPOPI! 🎄Our Board members and staff send warm wishes for a festive holiday season and a healthy, inspiring New Year. As 2025 comes to a close, we pause to reflect with gratitude on the dedication, collaboration, and innovation that have shaped our journey so far. 🎉May your holidays be filled with warmth, joy, and the company of family and friends, and may 2026 bring new opportunities for learning, connection, and progress together. #SeasonGreetings #IPOPI #NewYear2026

✨ @JohanPrevot, IPOPI's Executive Director, joined the High Level Meeting during the Policy Discussion session where was discussed one of the most urgent topics for the future of rare disease care: How can Europe build early access models that ensure equitable access to innovative orphan therapies and diagnostics? Moderated by @josepfigueras, Director Emeritus of the European Observatory on Health Systems and Policies, the panel brought together key political, clinical and patient-driven perspectives. A vital discussion on policy and funding — and on how Europe can finally close the gap between innovation and the patient. #HLMRARE2025

IPOPI invites you to the upcoming PID Forum at the European Parliament! PID Forum: When Rare Meets the Not-So-Rare – The Case of PIDs & SIDs Register here: forms.gle/eHJsMh7aoD1VP3… We are delighted to hold this session under the patronage of MEP @hadjipantela (EPP, Cyprus) and MEP @TomislavSokol (EPP, Croatia), with a video contribution from MEP @ignaziomarino (Greens, Italy). IPOPI will host a high-level roundtable exploring how expertise in primary immunodeficiencies (#PIDs) can enhance the understanding and management of secondary immunodeficiencies (#SIDs). We look forward to welcoming you!

📖 What’s new in our autumn newsletter? The 2024 PIDetect Programme is empowering healthcare professionals across Mali and improving PID diagnosis nationwide. Find out how these efforts are creating real impact and what’s next! ✨ Read more: e-news.ipopi.org #PIDetect #PrimaryImmunodeficiency #Mali

🎥 Discover Pulmonary Alveolar Proteinosis, a rare but serious complication affecting patients with primary immunodeficiencies. Our upcoming Mini-Doc sheds light on this hidden challenge, with expert insights, clear explanations, and the importance of early diagnosis. 🔔 Coming soon — turn on notifications so you don’t miss it! Check it here: youtu.be/VogGge5OtIk #IPOPI #MiniDocs #PAP #PIDs

An important topic- congrats again to Inês Serra!

AMR is a major threat — especially for people with PIDs, who rely on effective antibiotics throughout life. As ENIPAR members, IPOPI echoes concerns raised during #WorldAMRWeek: uneven national action, outdated AMR plans and unequal access to diagnostics. We call for stronger national strategies, better training and improved infection prevention. 📄 ENIPAR Call to Action: ehma.org/app/uploads/20… #AMR #PID #WAW

Have you read our Autumn Newsletter? 🍂 📬 We shared an important milestone in neonatal screening — a major step forward for early diagnosis and improved patient care. 🔗 Missed it? Catch up on the full newsletter here: e-news.ipopi.org #IPOPI #SeasonalNewsletter

Patient communities must not be excluded from rare disease research – An insightful op-ed by MEPs @JerkovicRomana and @nicogoncas. Just published in The Parliament Magazine, this article emphasizes the critical need for patient-centred research in the rare disease landscape. As co-hosts of the last PID Forum on patient-centred research, they bring forward a powerful message on the importance of including patient voices in shaping research agendas. Their call to action aligns with IPOPI’s mission to ensure that rare disease communities are not only heard but actively involved in the research process. We’re proud to have had the opportunity to collaborate with these MEPs to advocate for more inclusive and impactful research that addresses the real needs of patients living with primary immunodeficiencies (PID). 💬 "Patients must not be sidelined – their perspectives and experiences are vital in creating better treatments and improving lives." Read the full article and join the conversation🔗 theparliamentmagazine.eu/news/article/o… #RareDisease #PatientCentredResearch #PID #IPOPI #EUHealthPolicy #InclusionMatters #RareDiseases

IPOPI welcomes the positive CHMP opinion recommending EU marketing authorisation for Waskyra™, a groundbreaking ex vivo gene therapy for Wiskott-Aldrich Syndrome. “This is a major milestone for gene therapy in the EU,” said @JohanPrevot, IPOPI’s Executive Director. “It brings new hope for WAS patients and paves the way for more innovative treatments,” added IPOPI President @martinepergent.

Can the new Critical Medicines Act (CMA) truly make a difference for patients? In a new EURACTIV op-ed, IPOPI President @martinepergent and Executive Director @JohanPrevot share key patient priorities for the CMA and the long-term changes it could bring for people living with primary immunodeficiencies (PIDs) 📖 Read the full article on: euractiv.com/opinion/the-cr…