World Orphan Drug Congress USA

?" Sarepta Therapeutics and Arrowhead Pharmaceuticals teamed up on an siRNA partnership that has one of the biggest promises of biobucks in recent years. The deal, announced Tuesday morning, will see Sarepta snag the rights to up to 13 pipeline programs, including four already in the clinic, that are primarily focused on #rarediseases. In exchange, Sarepta will pay $500 million in upfront cash and $325 million in an equity investment at a 35% premium." World Orphan Drug Congress USA https://lnkd.in/euyUGMSg

?? We are excited to announce World Orphan Drug Congress USA's 2025 Media Partner, CheckRare!? CheckRare?is the only publisher and learning platform focused exclusively on #rarediseases and #rarecancers –?bringing physicians and patients the best science, research, and clinical practice information and present it in understandable and clinically useful formats that inform health care delivery and improve patient outcomes. ?? ???? At CheckRare, they recognize all rare disease definitions –?establishing?their platform to share differing perspectives and raise awareness of rare diseases. They believe that to get the full story on any rare disease, they need to understand the illness from the patients’ and physicians’ perspectives, as well as industry and regulatory viewpoints. With incredible partners, like CheckRare, covering our 2025 edition – you don't want to miss out. ??? ???????? $??,?????? ???? ???????? ???????????? with our new 60% early-bird discount here: https://lnkd.in/eaz-cxwZ Patient advocates and hospitals – ???????????? ???????? ???????? ???????? today: https://lnkd.in/ezqcD2G4 #WorldOrphanUSA #OrphanDrugs #RareCare #PatientAdvocacy #PatientCare

"Patients with #rarediseases often experience prolonged diagnostic delays. Ordering appropriate genetic tests is crucial yet challenging, especially for general pediatricians without genetic expertise...the study aims to develop a machine learning model (Phen2Test) that uses patient phenotypes documented in electronic health records (EHRs) to predict whether an experienced specialist will directly order ES/GS, bypassing the need for a gene panel test." World Orphan Drug Congress USA https://lnkd.in/e2HSPVfH

We are officially just 5?? months away from the 2025 edition of the?World Orphan Drug Congress USA! As our countdown continues, we're thrilled to highlight this thought-provoking, women-led panel discussion that addresses a vital topic in rare disease care ?? ??????????: ?????????????????? ???? ???????????????? ???????????? ?????? ?????????????? ???????????????? ???? ?????????????? ???????????? ?????? ?????????????????????????????????? (?????????? ??????????) ? Moderator: Raquel Cabo, Head of Patient Advocacy, Communications, Commercial Strategy, Ovid Therapeutics ? Melissa Penn, JD, MPH, Director, Patient Engagement R&D, Bayer ? Deborah Requesens, Founder, SHER (Hispanic Society Rare Diseases) ? Jenifer Ngo Waldrop, Executive Director, Rare Disease Diversity Coalition, Black Women's Health Imperative Together, our panelists will explore actionable strategies to enhance diversity, equity, and inclusion throughout the clinical trial process and beyond. You don’t want to?miss the opportunity to be part of the conversation shaping the future of rare disease treatment and access. ???? ?? ?? Mark your calendars for this must-attend session and ???????????? ???????? ???????? ???? ?? ???????????????????? ???????? ??????????:?https://lnkd.in/eaz-cxwZ Patient advocates and hospitals, ?????????? ???????? ???????? ??????-???????????? ???????? ????????: https://lnkd.in/ezqcD2G4 ?????????????? ???? ??????????: https://lnkd.in/ev8pfujX ??? April 22-24, 2025 ?? Boston Convention & Exhibition Center #WorldOrphanUSA #RareDisease #OrphanDrugs #ClinicalTrials #PatientAdvocacy #PatientCare

Excited to announce that I will be presenting at the World Orphan Drug Congress USA 2025! ?? As the CEO of an AI Startup, I’ll be sharing insights on “Unlocking the Future: The Convergence of AI, Personalized Medicine, and Genomics.” This session will explore how cutting-edge technologies like AI are transforming the landscape of personalized healthcare, enabling innovative solutions for rare diseases, and shaping the future of genomics. I look forward to connecting with thought leaders, innovators, and advocates dedicated to making an impact in the world of orphan drugs and beyond. Let’s work together to drive progress for patients and the global healthcare community. If you're attending the Congress, I’d love to see you there! Feel free to reach out and connect to discuss how we can collaborate to advance this critical work. #AI #HealthcareInnovation #PersonalizedMedicine #Genomics #OrphanDrugs #WorldOrphanDrugCongress2025 #Leadership #RareDiseases

Meet the inspiring women shaping the future of patient advocacy?at?#WorldOrphanUSA 2025! ???? We’re honored to welcome speakers from organizations dedicated to empowering patients and driving transformative change in the rare disease community, including: ? Jean S., Executive Director, Genetic ALS & FTD: End the Legacy ? Sophia Zilber ??,?Board Member, Patient Registry Director,?Cure Mito Foundation ? Mackenzie Abramson, MPH,?Patient Advocate and Sr. Manager of Research Program Communications,?Global Genes ? Heidi Edwards, President, Sisters' Hope Foundation ? Allison Peck, CFO, Cure VCP Disease ? Annette Bakker, President, Children's Tumor Foundation ? Sonia Gobeil,?Co-Founder,?Ataxia of Charlevoix-Saguenay Foundation ? Pangkong Fox,?Science Engagement Director,?CACNA1A Foundation Hear their powerful stories, innovative strategies, and insights on improving patient care, access, and empowerment on the World Orphan Drug Congress USA stage April 22-24. Together, we’ll explore how to amplify patient voices and address unmet needs across the globe. ?? ?? Join us in making a difference –?register now and be part of the movement. ? ?????? ????% ?????? ?????????????? ???????????? ?????? ??????????-???????? ???????? ?????????????? ???? ???????? ???? ??????????: https://lnkd.in/eaz-cxwZ ? ???????? ???????????? ?????? ?????????????? ???????????????????? & ?????????????????? ????????: https://lnkd.in/ezqcD2G4 ??? Interested in sharing you story or expertise? ?????????????? ???? ?????????? ??????????: https://lnkd.in/ev8pfujX #RareDisease #OrphanDrugs #PatientCare #PatientAdvocacy

?? We are excited to welcome back our Silver Sponsor,?AnovoRx, to the 2025 edition of the World Orphan Drug Congress USA! ?????????? ???????? ????????????????? ???????????? ?????????????? ?? Dedicated. Exclusive. Unique. Every orphan product requires its own solution, and only Anovo is built from the ground up to develop and deliver the optimal solution, every time. Their business is entirely dedicated to supporting orphan products, and they only work as the exclusive service provider for those products – providing integrated, customized program management, 3PL, Specialty Pharmacy, and Patient Services, supported by one management team. This makes them unique, enabling the solutions they implement to be extraordinarily successful – measured by client satisfaction, patient satisfaction, speed-to-market, successful reimbursement, patient retention, positive health outcomes, and more. Connect with the AnovoRx team April 22-24 at the Boston Convention & Exhibition Center April 22-24! ??? Secure your #WorldOrphanUSA ticket at the lowest rate possible and ???????? $??,?????? with our early-bird discount. Don't wait, this ?????????? ?????????????? ???????????? ?????? ???????????? ???????????? ????????. ?????? ?????????? ?????? ?? https://lnkd.in/eaz-cxwZ Patient advocacies and hospitals, claim your ???????? ???????? here ?? https://lnkd.in/ezqcD2G4 ?? Interested in exhibiting alongside the biggest companies in the #raredisease and #orphandrug space? Reserve your spot – ?????????????? ???? ?????????????? ?????????? ?? https://lnkd.in/ev8pfujX ?? Download our sponsorship brochure to ?????????? ???????? ?????????? ?????????????????? ?????????????????????????? ???? ?????? ???????????????? ?? https://lnkd.in/eWMGyc3j

"National Center for Advancing Translational Sciences (NCATS) is developing new approaches to diagnose and treat people with #rarediseases more quickly through resources, capabilities, and funding." ?? Understanding rare disease biology and therapeutic targets, especially common features ?? Building platform approaches that advance drug discovery and development efforts for many diseases ?? De-risking promising new treatments so they’re more attractive for commercial development ?? Leveraging new technologies, such as AI/machine learning and whole-genome sequencing, to improve diagnostic approaches ?? Modernizing clinical trial approaches for gene therapies and other newer treatment modalities ?? Bringing partners together to ensure health solutions reach the people who need them the most World Orphan Drug Congress USA https://lnkd.in/eAYcFdGN

Did you know we have ???????? ?????? ?????????????? ?????????????????? for the 2025 edition of World Orphan Drug Congress USA?! ?? ?? Industry experts will take the?#WorldOrphanUSA?stage April 22-24 to cover the latest and most prevalent topics in the #orphandrug and #raredisease space – including digital health and artificial intelligence, clinical development and regulatory, discovery, patient data, global market and patient access, diagnosis, and more. ??? ???????????? ?????? ??????+ ???????????????? ????????:?https://lnkd.in/e-BZhAYr Plus, check out some speaker session highlights from our agenda release earlier this week! ?? ????????????????????????: ?????????????? ?????????????? ???????? ?????????????????? – ???????????? ?????????? -?Diego F Guarin MD MPH MSc, Executive Director, Regional Market Access, Latin America,?Merck ??????????: ?????????????????? ?????? ????????????: ?????? ?????????????????????? ???? ????, ???????????????????????? ????????????????, ?????? ???????????????? - Moderator:?Amy Nicole Nayar PharmD, HEOR, Vice President of U.S. Patient Advocacy & Government Affairs,?Novartis Gene Therapies -?Guadalupe Hayes-Mota, CEO & Co-founder,?Stealth Startup?& Senior Lecturer,?Massachusetts Institute of Technology -?Durhane Wong-Rieger, President & CEO,?Canadian Organization for Rare Disorders - J?rg Schüttrumpf, M.D., Chief Scientific Innovation Officer,?Grifols ????????????????????????: ???? ?????? ???????????????????? ???? ????????????????????: ???????? ?????????????????? ?????? ???????????? ???????????? ?????? ???????????????????? ?????????????????? - Swami Subramanian, Head US/Global Pricing,?Regeneron Don't forget to register with code AGENDA25 to ???????? ???????? $??,?????? ???? ???????? ????????????. ??? Secure your savings today before this offer expires tomorrow: https://lnkd.in/eaz-cxwZ *???????????????? ???????? ???????????????? ????????????, ???????????????? 15 ?? Inquire to speak alongside industry leaders: https://lnkd.in/ev8pfujX

The wait is finally over… the World Orphan Drug Congress USA 2025?conference agenda is officially LIVE! ?? Featuring the most distinguished leaders across the rare disease and orphan drug space, this year's?all-encompassing agenda is filled with more inspiring, industry-leading speakers than ever before, including: ? Deputy Director of the National Center for Advancing Translational Sciences (NCATS) Division of Rare Diseases Research Innovation,?The National Institutes of Health ? Chief Executive Officer, National Organization for Rare Disorders (NORD) ? Senior Director - Gene Therapy Program and Office of Emerging Medical Discoveries,?Boston Children's Hospital ? Patient Advocates ? President, Chief Executive Officer, Canadian Organization for Rare Disorders & RARE DISEASES INTERNATIONAL ? Vice President, Global Head Patient Experience & Insights,?Alexion Pharmaceuticals, Inc. ? Director, Center for Biologics Evaluation and Research,?FDA ? Vice President of Global Head Market Access Cell and Gene Therapy,?Bayer ? Secretary General,?EUCOPE - European Confederation of Pharmaceutical Entrepreneurs ? Vice President of U.S. Patient Advocacy & Government Affairs,?Novartis Gene Therapies ...?????? ???? ???????? ?????????????????????? ?????? ???????? ???????????? ????????: https://lnkd.in/enjrxJQd ? For a limited time, celebrate with us and book your ticket with code ???????????????? for an extra 10% off! That's ???????? $??,?????? ?????????? with our early-bird offer.? ??? Get your ticket at the ???????????? ???????? ???????????????? now: https://lnkd.in/eaz-cxwZ ??? ???????? ???????????? for patient advocates and hospitals available here: https://lnkd.in/ezqcD2G4 Interested in taking the stage at #WorldOrphanUSA 2025? ??? Share your inspiring story and showcase your expertise – ?????????????? ???? ?????????? ??????????: https://lnkd.in/ev8pfujX #RareDisease #OrphanDrugs #PatientAdvocacy #PatientCare