Thank you for the well-organized machinery that allowed us opportunities to arrange meetings. The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors and solution providers. Contribute to driving and directing the fast growing field of Health. Details of Biomarkers 2023 Conferences in Canada Biomarkers 2023 Your meeting registration confirmation will include a link to generate a customized letter of invitation, which can be used when applying for a visa. All are welcome to join the celebration to cheer on our skaters. Thank you to all the folks at NORD for your remarkable efforts to make this such a success.. With its high quality, it provides an exceptional value for students, academics and industry researchers. By continuing to use this website, we assume you agree to our, Click Here to Register or Modify Your Registration. Davide Zecchin and Sara Barbera Martin (Senior Post-Doctoral Research Associates, Kinsler lab, Francis Crick Institute), Prof Rob Semple, Professor of Translational Molecular Medicine, University of Edinburgh, What causes insulin resistance, and what can we do about it?, Prof Steve Hart, Professor in Molecular Genetics, UCL, Dr Helen Brittain, Clinical Lead for Rare Disease Diagnostics, Genomics England, "The 100,000 Genomes Project and beyond: An update on Rare Disease Diagnosis and Research", Prof Sergi Castellano, Professor of Genomics, UCL, Prof Hannah Mitchison, Professor of Molecular Medicine, UCL, "Rare genetic respiratory diseases and targeting genetic therapies to the airways", Prof Alan Warren, Professor of Haematology, University of Cambridge, "Convergent somatic evolution commences in utero in a germline ribosomopathy", Prof Veronica Kinsler, Professor of Paediatric Dermatology and Dermatogenetics, GOSH, UCL and the Francis Crick Institute, Maanasa Polubothu (clinical academic PI, UCL and consultant Great Ormond St) and Dale Bryant (senior post-doctoral research associates, Kinsler lab, Francis Crick Institute), Dr Antoine de Fougerolles, CEO Evox Therapeutics, "Exosome therapeutics: creating and enabling genetic medicines", Prof Mina Ryten, Professor of Clinical Genetics, GOSH and UCL, "Leveraging transcriptomics to understand rare genetic diseases of the human brain", Prof Siddharth Banka, Professor of Genomic Medicine and Rare Diseases, University of Manchester, "Mechanistic and clinical heterogeneity of single gene disorders illustrated by non-muscle actinopathies", Prof Paul Gissen, Professor of Paediatric Metabolic Diseases, GOSH and UCL, "Towards understanding a rare membrane trafficking disorder ARC", Prof Mariya Moosajee, Professor of Molecular Ophthalmology, Moorfields, UCL and the Francis Crick Institute, "Choroideremia - is it just a rare eye disease? This years conference will include presentations on next generation technology, long-read genome sequencing, patient empowerment and health equity, and more. Rare Diseases in the XXI Century Scientific Conference. October 1, 2022Deadline for submission of full-length manuscripts for peer-review in the Lysosomes issue of Molecular Genetics & Metabolism, February, 2023. Christines work at Cure GM1 has involved a broad range, including animal models, biomarkers, gene therapy, enzyme replacement therapy, patient registries, patient reported outcomes, newborn screening and the first-ever GM1 caregiver preferences study. 1140062 and a company registered in England and Wales no. By Hangin Out. Stay Informed With NORDs Email Newsletter, "Skate Under the Stars: A Rare Disease Celebration", "2023 Illinois Rare Disease Day at the Capital", "The 2nd Annual CHOC and UCI Rare Disease Symposium & Family Conference", "Building Bridges to the Future: Jamals Helping Hands Anniversary BBQ", "Rare Disease Innovation & Partnership Summit", Renaissance New Orleans Pere Marquette French Quarter, "2023 Rare Disease Day Symposium at the Frank H. Netter MD School of Medicine", Launching Registries & Natural History Studies, Skate Under the Stars: A Rare Disease Celebration, CHOC Rare Disease Grand Rounds: Pyridoxine Dependent Epilepsy (PDE), 2023 Illinois Rare Disease Day at the Capital, The 2nd Annual CHOC and UCI Rare Disease Symposium & Family Conference, Building Bridges to the Future: Jamals Helping Hands Anniversary BBQ, Rare Disease Innovation & Partnership Summit, 2023 Rare Disease Day Symposium at the Frank H. Netter MD School of Medicine. Read more: https://bit.ly/3tGXzXn, Read the @RareDiseases Summit 2022 Recap! WebThe joint event RE(ACT) Congress and IRDiRC Conference 2023 aims to bring together scientific leaders and experts and young scientists from various breakthrough scientific fields to present cutting-edge research, exchange ideas, and discuss rare diseases research policies. The virtual approach cant replace in-person conversations, but can reach people and enable important connections. Download the presentation, Maria KALOGEROPOULOU, Head Value Access, Health Policy & RWE, IQVIA Hellas 55 Kenosia Avenue The goals of Rare Disease Day at NIH are to: Rare Disease Day at NIH was held at NIH Main Campus (Natcher Conference Center) on Tuesday, Feb. 28, 2023, from 9 a.m. to 5 p.m. EST. Workshops, Public meeting: FDA Rare Disease Day 2023, An official website of the United States government, : NORD is not a medical provider or health care facility and thus can neither diagnose any disease or disorder nor endorse or recommend any specific medical treatments. Location: Bartosza Gowackiego 35, Lublin, Poland. Download the presentation, Panagiota MITROU, Deputy Head of the Autonomous Department of Therapeutic Protocols & Patient Registries, Ministry of Health Join us for three days in Washington for the most comprehensive program and inclusive gathering of rare disease stakeholders. Marriott Marquis in Washington, DC, NORD Leads the Way: 900+ Influential Rare Disease Stakeholders from FDA, Patient Organizations, and Industry Gather for #NORDSummit 2023. Phone: 203-263-9938 Copyright 2023 Hanson Wade | Design and site by Event Engine| Hanson Wade Limited is registered in England & Wales, number 06752216. With an expert speaking faculty devoted to bringing safer and more effective gene therapies to rare disease patients, key questions will be answered on how best the field can overcome regulatory, clinical, manufacturing and pricing bottlenecks to progress gene therapies into and through the clinic. Learn more about how you can attend this event or add it to your calendar. WebRare Disease Conference 2023 - Rare Disease Innovation & Partnership Summit informaconnect.com wAIHA Warriors is providing travel , Continue reading "wAIHA Warriors Annual Patient Meeting", Since 2015, the Frank H. Netter MD School of Medicine at Quinnipiac University has hosted the Rare Disease Day Symposium, providing an opportunity for patients, family members, clinicians, and researchers to share their stories, research and insights into the development of novel therapeutics. WebInternational Conference on Rare Diseases 2023. Applications for the 2023 PhD programme are open! Download the presentation, Mari MUREL, ERICA Project Manager Volunteer. Danbury, CT 06810 Terrapinn is proud to be a member of isla. WebThe 6th Annual Gene Therapy for Rare Disorders 2023 Summit remains devoted to showcasing the top case studies and strategic learnings from the past year. FDA Meetings, Conferences and Workshops, Recalls, Market Withdrawals and Safety Alerts, FDA Meetings, Conferences, and Workshops: Past Events, Public Calendar - Meetings With FDA Officials, Public meeting: FDA Rare Disease Day 2023 - 02/27/2023, RDD 2023 Meeting Packet Brochure 02-02-2023_final_508. Download the presentation, Antonis KATTAMIS, Professor of Pediatric Hematology-Oncology, President, Hellenic Society of Pediatric Hematology-Oncology Each year, WORLDSymposium hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. Present and discuss your plans, activities and products. Changing lives of those with rare disease. From: 09/19/2023 To: 09/20/2023. 39 Dalton St, If you would like to find out more about how we manage your personal information please see our privacy policy. With August came the green light for bluebird bios Zynteglo in the US, the first FDA approval for a gene therapy in over three years. T: (+1) 617 455 4188 If you have problems viewing PDF files, download the latest version of Adobe Reader, For language access assistance, contact the NCATS Public Information Officer, National Center for Advancing Translational Sciences (NCATS), 6701 Democracy Boulevard, Bethesda MD 20892-4874 301-594-8966, U.S. Department of Health & Human Services, Clinical and Translational Science Awards (CTSA) Program, Rare Diseases Clinical Research Network (RDCRN), Therapeutics for Rare and Neglected Diseases (TRND), Additional Rare Diseases Research and Initiatives, Patient/Community Engagement & Health Information, Genetic and Rare Diseases Information Center, NCATS Toolkit for Patient-Focused Therapy Development, National COVID Cohort Collaborative (N3C), About NCATS Role in the NIH HEAL Initiative, Accelerating the Translation of Novel Compounds Toward INDs for Subsequent Clinical Testing, Fiscal Year 2019 Funded Projects and Prizes, NIH HEAL Initiative Funding & Collaboration Opportunities Led by NCATS, NCATS Program-Specific Funding Information, Prior NIH Approval of Human Subjects Research Frequently Asked Questions, NCATS Challenges and Prize Competitions Program, Bias Detection Tools in Health Care Challenge, LitCoin Natural Language Processing (NLP) Challenge, NCATS Rare Diseases Are Not Rare! On October 17 and 18, the National Organization for Rare Disorders (NORD) hosted the Rare Diseases and Orphan Products Breakthrough Summit. Brain Injury Alliance of All attendees who registered for On Demand will receive an email with a link to access the On Demand platform starting Monday, February 27 until Friday, March 31, 2023. Sheraton Harbor Island, 1380 Harbor Island Dr, San Diego, CA 92101. Jaguar Health, Inc. With August came the green light for, As the gene therapy field continues to break records, there are still significant challenges to overcome relating to safety, efficacy, and accessibility. Hear from medical students on rare disease education for medical professionals. Appraise and qualify unmet needs from clinician users. Speakers Sponsors Media Centre Contact Us Register close 2023 Sponsors Included: Why attend? MA 02199 Web8th International Conference on Rare Diseases and Orphan Drugs Osaka, Japan November 13-14, 2023 6th International Conference on Tropical and Infectious Diseases Bali, Indonesia December 07-08, 2023 6th Pathology and Infectious Disease Conference Prague, Czech Republic December 14-15, 2023 13th European Epidemiology and Public Youll have opportunities to learn quickly, advance your career, and to meaningfully impact our customers and our business. The conference is a place to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies. Keep up-to-date on the latest NORD Summit news, Director of Development and Strategic Partnerships, The Myositis Association, Lois Vierk, President, National Eosinophilia Myalgia Syndrome Network, Susan Fernbach, RN, Director of Genetic Outreach, Baylor College of Medicine and member of NORDs Summit Advisory Committee. Annual Summit on Rare Diseases and Orphan Drugs, International Conference on Rare Infectious Diseases (ICRID), International Conference on Rare Diseases and Indigenous Genetics (ICRDIG), International Conference on Orphan Drugs for Rare Diseases (ICODRD), International Conference on Clinical Genetics and Rare Diseases (ICCGRD), International Conference on Orphan Drugs, Rare Diseases and Conditions (ICODRDC), International Conference on Orphan Drugs and Rare Diseases (ICODRD), International Conference on Rare Diseases and Orphan Drugs (ICRDOD), Creative Commons Attribution 4.0 International license. Event Details. How To Help A Family Member With Mental Illness, How School Affects Mental Health In High School Students, Empowering community members through mental health education, advocacy, and support, Effects of Financial Illiteracy on Physical, Mental, Emotional, Spiritual Health. July 1, 2022Abstract submission website open. Each year, Global Genes convenes one of the worlds largest gatherings of rare disease patients, caregivers, advocates, healthcare professionals, WebThis conference is a must attend event for thought leaders, advocates, researchers and industry executives trailblazing novel solution-driven pathways for the future of gene therapy and rare disease innovation. Discussions will focus on Learn more about how you can attend this event or add it to your calendar. Receive updates on the NORD Summits agenda, speakers, registration, and more by opting into our mailing list. FDA will host Rare Disease Day, a virtual public meeting, on February 27, 2023, 9:00 am 4:45 pm ET, in global observance of Rare Disease Week. E:info@hansonwade.com, Sheraton Boston Hotel FDA will host Rare Disease Day, a virtual public meeting, on February 27, 2023, 9:00 am 4:45 pm ET, in global observance of Rare Disease Week. Broadly acclaimed speakers, the most recent frameworks, methodologies, and the most current updates in this field are indications of this conference. THE BENEFITS PROVIDED TO THE ATTENDEES ARE: PARTICIPANTS - FIVE REASONS WHY I SHOULD ATTEND THE CONGRESS: PARTNERS - REASONS WHY I SHOULD EXHIBIT DURING THE CONGRESS: Copyright All rights reserved | Design & Developed by, Re-conceptualizing existing paradigms of diagnosis and difficulty, Breaking down silos how different models fit together. 4 min read. Applications for the 2023 PhD programme are now open until 12:00 noon (GMT) on Wednesday 22 March 2023. NORD, along with its more than 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services. Highlight NIH-supported rare diseases research and the development of diagnostics and treatments. Over 830 enthusiastic attendees from across the globe convened to address and take action on the opportunities and challenges facing the rare disease community. Vice President, Discovery and Translational Research. Dr. Marks Keynote Address: Taking Gene Therapy to the Next Level, was presented on Friday, February 24, 2023 at 7:30 AM EST, at the 19th Annual WORLDSymposium in Orlando, Florida. The. The conference will start at approximately 13:00 on Monday 24 April 2023 and close at approximately 14:30 on Wednesday 26 April 2023. 15 16 Mar, 2023 Onsite & Online , Meet us at the World Rare Disease Day Conference in Medina, Saudi Arabia, on March 15-16, 2023 to learn more about how we work together with patients, physicians, and pharma partners to deliver data-driven, life-changing answers in rare and neurodegenerative diseases. By App. Sessions will include information from patients, physicians, and researchers regarding wAIHA clinical trials, current treatments, access to care, federal and state advocacy efforts, etc. With a focus on the most critical topics, including accelerated approval, newborn screening, and equitable access to care, the 2023 NORD Summit promises a full program of productive discussions and collaborative solutions. We are a biomedical discoveryinstituteresearching the biologyunderlying human health. After reviewing numerous nominations, and considering many amazing individuals, the WORLDSymposium2023 Awards Committee has selectedWilliam A. Gahl, MD, PhD, as the recipient of the2023 Roscoe O. Brady Award. Engage with the FDA to provide your perspectives as a patient, caregiver or family member. The World Orphan Drug Congress USA is a place where innovation and expertise is showcased, solutions are found and learning is done. Rare Disease Day at NIH will be held in person at NIH Main Campus (Natcher Conference Center) on Tuesday, Feb. 28, 2023, from 9 a.m. to 5 p.m. CME/CE/CEU credits are available for for select On Demand Scientific and Satellite Sessions. February 27 @ 9:00 am - 4:45 pm EST. WebHome | Leading Rare Diseases Conference | Orphan Disease Conferences | Orphan-Drugs Meetings May 25-26, 2023 10:00 AM GMT London, UK 4th International Conference on Rare Diseases and Orphan Drugs Pioneering Breakthroughs on Rare Diseases 83 : days 02 : Hours 59 : Mins 55 Secs Brochure Download Advertising Webinars Come face to face with life-changing science, Engage and inspire with discovery science, The latest science and stories from the Crick, Working to discover the biology of health and disease. I was able to watch a little in person and then I was able to access NORDs session files. WORLDSymposium is excited to announce the return of Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration, as the 2023 Keynote Speaker. WORLD is an acronym that stands for Were Organizing Research on Lysosomal Diseases. 1779 Massachusetts Avenue The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely. Please enable JavaScript in your web browser to get the best experience. Click here to access the Guide for Authors or to Submit Your Paper. NORD, a 501(c)(3) organization, is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. May 23-25, 2023 Gaylord National Resort & Convention Center Washington, D.C. Join the webcast to watch the livestream on February 27 beginning at 9 a.m. (ET). This years The Second Annual CHOC and UCI Rare Disease Symposium & Family Conference will bring together over 100 advocates, researchers, clinicians, students, and families to share research, knowledge, experience on Rare Diseases. Thank you for the opportunity to present on the vitally important topic of transition of care., NEMSNs Board thanks you very kindly for your scholarship to our organization to attend the 2020 Rare Summit. , Continue reading "Skate Under the Stars: A Rare Disease Celebration". WORLDSymposium and the Lysosomal Disease Network (LDN) are separate and independent entities and are not affiliated in any way. Rare Disease Conferences 2023/2024/2025 is an indexed listing of upcoming meetings, seminars, congresses, workshops, programs, continuing CME courses, trainings, summits, and weekly, annual or monthly symposiums. How do lay professionals deal with issues around gender and sexuality in the community? August 1, 2022Registration for the conference opens. International Conference Rare Diseases and its Treatment aims to bring together leading academic scientists, researchers and research scholars to exchange and share their experiences and research results on all aspects of Rare Diseases . This free family-friendly BBQ will feature live music, a kid's zone, food trucks, educational resources, and more! Phone: 617-249-7300, Danbury, CT office (February 28, 2023) About Rare Disease Day Launched by EURORDIS-Rare Diseases Europe in 2008, Rare Disease Day is the patient-led international awareness campaign that brings people together in solidarity with the 300 million patients impacted Initiate a mutually beneficial dialogue among the rare diseases community. Boston Summary http://ow.ly/5Wzs50Lj6CJ #NORDSummit. This exciting United States. Copyright 2023 NORD National Organization for Rare Disorders, Inc. All rights reserved. WORLDSymposiumwas pleased to announce the recipients of the 2023 Young Investigator Award. Illinois Rare Disease Day at the capital brings together rare disease advocates from across the state to make , Continue reading "2023 Illinois Rare Disease Day at the Capital". Roscoe O. Brady Award Speaker: William A. Gahl, MD, PhD , Basic Science, Translational Research, ClinicalApplications, Contemporary Forum, and Late-Breaking Sessions, Emerging Trends: State-of the-Art for Experts, 2023 Scientific Sessions, ePosters and Satellite Symposia are available On Demand until. About Rare Disease Day . This website uses cookies to improve your experience. WebThe joint event RE(ACT) Congress and IRDiRC Conference 2023 aims to bring together scientific leaders and experts and young scientists from various breakthrough scientific ContactOOPDOrphanEvents@fda.hhs.gov | 301-796-7634 Overview, new treatments, and the potential for Newborn Screening for Pyridoxine Dependent Epilepsy (PDE) Speaker: Curtis R. Coughlin II, PhD, MS, MBE CHOC Grand Rounds is part of the CHOC UCI Rare Disease Day. Learn more about DRDRI and NCATS' rare diseases research programs and access shareable resources to help raise awareness about rare diseases. The theme for the conference this year is Bio Markers: Future of Medical Diagnosis and Treatment . Join the National Organization for Rare Disorders (NORD) October 15-17, 2023 for the Rare Diseases and Orphan Products Breakthrough Summit. Each year, WORLDSymposiumrecognizes one individual for innovation and accomplishment in the field of lysosomal disease research and therapy. The Francis Crick Institute1 Midland RoadLondon NW1 1AT, View upcoming changes to normal opening times. Session: R&D Spotlight - Rare Disease Date: Tuesday, April 25, 2023 Time: 4:00 p.m. - 5:15 p.m. Greenwich Mean Time Venue: ETC Venues, 155 Bishopsgate, London, England EC2M 3YD About BioTrinity BioTrinity is a London conference that catalyzes growth and supports in-person re-engagement across the life sciences industry. November 15, 2022Notification of abstract acceptance for poster or platform presentation at WORLDSymposium 2023. Participants can join in on a virtual walk/run to raise awareness for Rare disease day (wearing stripes!). We are always looking for ambitious people to join our team. Leveraging the momentum for a comprehensive rare disease strategy Introductory Speech: An (February 28, 2023) About Rare Disease Day Launched by EURORDIS-Rare Hear directly from the FDA on initiatives to advance medical product development for rare diseases. Join the National Organization for Rare Disorders (NORD) October 15-17, 2023 for the Rare Diseases and Orphan Products Breakthrough Summit. Third International Summit on Human Genome Editing Previous Events; Today Next Events; 1st International UAE Rare Disease Society Congress Feb 28 February 28 - March 1. October 1, 2022Deadline for abstract submission to WORLDSymposium 2023. We are looking forward to gathering in person for Rare Disease Day 2023 on February 25th starting at 9:00 am at the Prince Conference Center (Grand The .gov means its official.Federal government websites often end in .gov or .mil. Conferences, and WebThe conference is a place to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies. All Info for H.Res.181 - 118th Congress (2023-2024): Expressing support for the designation of February 28, 2023, as "Rare Disease Day". Demonstrate the NIH commitment to helping people with rare diseases through research. It also provides a premier interdisciplinary platform for researchers, practitioners and educators to present and discuss the most recent innovations, trends, and concerns as well as practical challenges encountered and solutions adopted in the fields of Rare Diseases. Read full announcement here. Click here to access the Guide for Authors or to Submit Your Paper. All Info for H.Res.181 - 118th Congress (2023-2024): Expressing support for the designation of February 28, 2023, as "Rare Disease Day". How are you raising awareness for the rare community this Rare Disease Day? Planning committee members included representatives from the following organizations: Progress in data science and an increased understanding of disease genetics lead experts to agree that more than an estimated 10,000 rare diseases are affecting about 30 million people in the United States. Download the presentation, International Conference on Rare Diseases 2023, Leveraging the momentum for a comprehensive rare disease strategy. Visit our exhibition on genome editing from 11 February 2023. To register for On Demand, click here, and then click Already Registered? to modify your registration or click Register to access this content. (February 28, 2023) About Rare Disease Day Launched by EURORDIS-Rare Diseases Europe in 2008, Rare Disease Day is the patient-led international awareness campaign that brings people together in solidarity with the 300 million patients impacted So you can: Exchange ideas, Build brand, Form friendships and partnerships, Grow your professional network, Explore the future, Uncover new opportunities., ADVANCED THERAPIES - CLINICAL DEVELOPMENT. WebAll MassBio events adhere to our conference Diversity Policy and Anti-Harassment Policy. This year conference will comprises of major sessions designed to offer comprehensive sessions that address current issues in various field of RARE DISEASES. Rare Disease Conferences 2023/2024/2025 will bring speakers from Asia, Africa, North America, South America, Antarctica, Europe, and Australia. We send our appreciation!, It was a fantastic conference and well organized; I have only heard positive comments! Following last year's inaugural meeting, we are very pleased to be hosting the second Crick Rare Diseases Conference to be held at the Crick on 28 February to mark World Rare Diseases Day 2023. Google Calendar. Rare Disease Therapies Development IL, Virtual Summit. Suite 310 February 21, 2023: Robert J. Gorlin Symposium and Emerging Trends February 22-26, 2023: Research Meeting Hilton Orlando, Orlando, FL, USA Scientific Read full announcement here. Presentations. Google Calendar iCalendar Outlook 365 Outlook Live Details Date: February 27 Time: 9:00 am - 4:45 pm EST Event Category: NORD Sponsored Rare Disease Day Event Website: NORD at UVA Speaker Panel 2023 . February 21, 2023: Robert J. Gorlin Symposium and Emerging Trends, Hilton Orlando6001 Destination ParkwayOrlando Florida, USA, 32819Phone: +1-407-313-4300Website: hilton.comWebsite: thehiltonorlando.com. Prof Veronica Kinsler, Professor of Paediatric Dermatology and Dermatogenetics, Paul Nioi, Vice President, Discovery and Translational Research. The site is secure. November 18, 2022 Registration is now open for the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference focusing on neuromuscular diseases, including Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), myasthenia gravis, and Pompe disease, among others. Join us virtually or in-person on March 10 for the 10th Annual Rare Disease Symposium. 13 - 14 September 2023 Princeton, New Jersey Register Why attend? Translate technological capabilities into clinical applications, relevant to daily practice. NCATS is committed to using research to address the public health crisis presented by rare diseases. The conference seeks to contribute to presenting novel research results in all aspects of Rare Diseases and Orphan Drugs. Shine a spotlight on stories told by people living with a rare disease, their families and their communities. February 13, 2023 Speaker and Abstract Submissions Open, March 10, 2023 Speaker Submission Deadline, March 15, 2023 Scholarship Application Window Opens, September 5, 2023 Early Bird Registration Closes, July 14, 2023 Abstract Submission Deadline, August 15, 2023 Abstract Decision Notifications by NORD, September 22, 2023 Hotel Room Block Closes, October 15, 2023 NORD Rare Diseases & Orphan Products Breakthrough Summit Welcome Reception, October 16 -17, 2023 NORD Rare Diseases & Orphan Products Breakthrough Summit, What an incredible lineup of speakers. Download the presentation, Magda CHLEBUS, Executive Director, Science Policy & Regulatory Affairs At EFPIA Advancing Rare Disease Research with Patient Centrality and Precompetitive Approaches- Screen4Care as a Case. Congratulations to Christine Waggoner, the recipient of the WORLDSymposium 2023 Patient Advocate Leader (PAL) Award. People loved the speakers, the timely topics, the exhibit hall, and networking opportunities. The Lysosomal Disease Network (U54NS065768) is a part of the National Institutes of Health (NIH) Rare Diseases Clinical Research Network (RDCRN), supported through collaboration between the NIH Office of Rare Diseases Research (ORDR) at the National Center for Advancing Translational Science (NCATS), the National Institute of Neurological Disorders and Stroke (NINDS) and National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). Stakeholders are invited to provide their perspectives on the discussion questions through the public docket.