Distribution of exhibitor prospectus and support opportunities to all prior supporting companies, and also available by request. 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. I was able to watch a little in person and then I was able to access NORDs session files. WORLDSymposium is managed by GMI, Inc. and Saterdalen & Associates, LLC. 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. 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. Identify pertinent evolution of Health technologies in RARE DISEASES fields. October 15-17, 2023 The Francis Crick Institute Limited is a registered charity in England and Wales no. Event Details. May 23-25, 2023 Gaylord National Resort & Convention Center Washington, D.C. The International Research Conference Aims and Objectives. The event will be shared in multiple Facebook support groups for individuals specifically with PKU. 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. How competent are lay professionals in addressing womens issues? Details of Biomarkers 2023 Conferences in Canada Biomarkers 2023 Read full announcement here. WebWorld Congress on Rare Diseases - 2023 +91 83102 90512 info@worldcongressonrarediseases.com About Conference The International Research Since 2011, NCATS and the NIH Clinical Center have sponsored Rare Disease Day at NIH as part of this global observance. Led by 60+ expert speakers and together with your peers, , Continue reading "Rare Disease Innovation & Partnership Summit", The first ever wAIHA Warriors Annual Patient Meeting will take place from March 24-26, 2023, in New Orleans. 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. Youll have opportunities to learn quickly, advance your career, and to meaningfully impact our customers and our business. Rare Disease conference listings are indexed in scientific databases like Google Scholar, Semantic Scholar, Zenedo, OpenAIRE, EBSCO, BASE, WorldCAT, Sherpa/RoMEO, Compendex, Elsevier, Scopus, Thomson Reuters (Web of Science), RCSI Library, UGC Approved Journals, ACM, CAS, ACTA, CASSI, ISI, SCI, ESCI, SCIE, Springer, Wiley, Taylor Francis, and The Science Citation Index (SCI). Summary. WebRare Disease Conferences 2023 2024 2025 is for the researchers, scientists, scholars, engineers, academic, scientific and university practitioners to present research activities Demonstrate the NIH commitment to helping people with rare diseases through research. Presentations. (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 Rare diseases often are difficult to diagnose it can take years. Stakeholders are invited to provide their perspectives on the discussion questions through the public docket. WebConference Series LLC Ltd welcomes you to attend the Biomarkers, Cancer Therapy & Clinical Research Conference to be held in Vancouver, Canada on September 23-24, 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. Highlight NIH-supported rare diseases research and the development of diagnostics and treatments. 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: This exciting event brings together over 900 rare disease advocates, experts, and stakeholders from around the world to tackle the most pressing issues facing the rare disease community. This years theme is Intersections with Rare Diseases A patient focused event. Participants will have the unique opportunity to: Public Docket Applications for the 2023 PhD programme are now open until 12:00 noon (GMT) on Wednesday 22 March 2023. Visit our exhibition on genome editing from 11 February 2023. All comments must be identified with the docket number FDA-2022-N-3072-0001 through April 7, 2023. Fax: 203-263-9938, Washington, DC Office 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. NCATS is committed to using research to address the public health crisis presented by rare diseases. Our mission is to promote inter-professional healthcare education and awareness of and , Continue reading "2023 Rare Disease Day Symposium at the Frank H. Netter MD School of Medicine", 1900 Crown Colony Drive By continuing to use this website, we assume you agree to our, Click Here to Register or Modify Your Registration. This conference is aimed at scientists, clinician scientists and doctors. We have kicked off 2023 with our continued support of Medics4RareDiseases (M4RD) during their annual symposium, and today, the emotive WORLDSymposiac/o GMI1900 NW Corporate BlvdSuite 410WBoca Raton, FL 33431. Download the presentation, International Conference on Rare Diseases 2023, Leveraging the momentum for a comprehensive rare disease strategy. 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. Shine a spotlight on stories told by people living with a rare disease, their families and their communities. Information on how to claim education credits is given only to registered attendees and is available in the 2023 WORLDSymposium mobile app. MA 02199 WebMENA Organization for Rare Diseases and UAE Genetic Diseases Association will conduct the MENA Organization for Rare Diseases Annual Meeting 2023 in Dubai from 3 to 5 The WORLD CONGRESS ON RARE DISEASES - 2023, - An International Conference by BioGenesis Health Cluster is among the Worlds leading RARE DISEASE Conferences. Speakers Sponsors Media Centre Contact Us Register close 2023 Sponsors Included: Why attend? WebThe conference is a place to meet and brainstorm ways to advance orphan drug development and improve access to life-saving therapies. E:info@hansonwade.com, Sheraton Boston Hotel WebRare Diseases Meet 2023 provides a premier interdisciplinary platform for researchers to present the latest research findings and describe emerging technologies, and directions in rare diseases and orphan drugs issues. 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 Event is from 2:00pm -4:00 pm There is no cost to participants to attend this event One of the greatest challenges individuals living with a rare disease, their families, and patient advocates have is finding and accessing information , Join the Jamals Helping Hands (JHH) five-year anniversary, Building Bridges to the Future, honoring their community, resilience, and strength. iBIO invites you to join patients, families, caregivers and other rare disease community members at this important event to educate Illinois legislators and the general public on the challenges faced by rare disease patients and their families. 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 NORD at UVA Speaker Panel 2023 . 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. Appraise and qualify unmet needs from clinician users. October 1, 2022Deadline for submission of full-length manuscripts for peer-review in the Lysosomes issue of Molecular Genetics & Metabolism, February, 2023. Webcast Information Most of these people are children. 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 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. Changing lives of those with rare disease. 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. Phone: 203-263-9938 In all, nearly 10% of the U.S. population have a rare disease. Location: Bartosza Gowackiego 35, Lublin, Poland. Overseas, July and August brought EMA approvals for PTC Therapeutics'Upstaza and BioMarins Roctavian, the first gene therapies for AADC deficiency and Haemophilia A respectively. 2022 has been a landmark year for the global gene therapy space. Rare Disease Day takes place worldwide, typically on or near the last day of February each year, to raise awareness among policymakers and the public about rare diseases and their impact on patients lives. Translate technological capabilities into clinical applications, relevant to daily practice. The conference seeks to contribute to presenting novel research results in all aspects of Rare Diseases and Orphan Drugs. Initiate a mutually beneficial dialogue among the rare diseases community. Leveraging the momentum for a comprehensive rare disease strategy Introductory Speech: An Hanson Wade's goal is to accelerate progress within organisations and across industries. Congratulations to this years recipients! Exchange the latest rare diseases information with stakeholders to advance research and therapeutic efforts. Download the presentation, Magda CHLEBUS, Executive Director, Science Policy & Regulatory Affairs At EFPIA WebThe Second Annual CHOC and UCI Rare Disease Symposium & Family Conference will bring together over 100 advocates, researchers, clinicians, students, and families to 39 Dalton St, This years conference will include presentations on next generation technology, long-read genome sequencing, patient empowerment and health equity, and more. Danbury, CT 06810 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. WebInternational Conference on Rare Diseases 2023. This symposium is designed to help researchers and clinicians to better manage and understand diagnostic options for patients with lysosomal diseases, identify areas requiring additional basic and clinical research, public policy and regulatory attention, and identify the latest findings in the natural history of lysosomal diseases. The. Dr. Gahl isthe Director of the Undiagnosed Diseases Program, a Senior Investigator in the Medical Genetics Branch and the Head of the Human Biochemical Genetics Section of the National Human Genome Research Institute (NHGRI). 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. WebThe 2023 Gordon Research conference on Lysosomal Diseases will cover the latest discoveries that advance knowledge about basic lysosomal function; how exactly function is impaired in lysosomal diseases - and how they inform more universal application in Receive updates on the NORD Summits agenda, speakers, registration, and more by opting into our mailing list. This website uses cookies to improve your experience. 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 This organization dedicated to bringing together a significant number of diverse scholarly events for presentation within the conference program. NORD is a registered 501(c)(3) charity organization. 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). Jaguar Health, Inc. Each year, WORLDSymposiumrecognizes one individual for innovation and accomplishment in the field of lysosomal disease research and therapy. February 27 @ 9:00 am - 4:45 pm EST. 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. 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. Brain Injury Alliance of Overcome Regulatory, Clinical, Manufacturing & Pricing Bottlenecks to Progress Safer, Efficacious, Accessible Rare Gene Therapies From Early Clinical Development Through to Approval, 2022 has been a landmark year for the global gene therapy space. Before sharing sensitive information, make sure you're on a federal government site. How are you raising awareness for the rare community this Rare Disease Day? This free family-friendly BBQ will feature live music, a kid's zone, food trucks, educational resources, and more! Contribute to driving and directing the fast growing field of Health. Save the date for NORD Summit 2023, set for October 16-17, 2023 in Washington, DC! (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 With Copyright 2023 NORD National Organization for Rare Disorders, Inc. All rights reserved. The conference will start at approximately 13:00 on Monday 24 April 2023 and close at approximately 14:30 on Wednesday 26 April 2023. Join our mailing list to receive exclusive content and offers. Assess and understand application challenges with current technology features. 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. 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. Registration is required. People loved the speakers, the timely topics, the exhibit hall, and networking opportunities. Attendees should look forward to coming together to celebrate five years of JHH successes and to build bridges to , Continue reading "Building Bridges to the Future: Jamals Helping Hands Anniversary BBQ", The Rare Disease Innovation & Partnership Summit, being held March 21-23 in Philadelphia, brings together experts from across the rare disease community to unite in areas of unmet medical need, create life-transforming therapies and breakthroughs, drive therapeutic progress, propel commercial strategies and inspire impactful advocacy. Suite 310 2018 Challenge Details, NCATS Rare Diseases Are Not Rare! 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". Events will run over a span of time during the conference depending on the number and length of the presentations. Registered Office: Eastcastle House, 27/28 Eastcastle Street, London, W1W 8DH. All Info for H.Res.181 - 118th Congress (2023-2024): Expressing support for the designation of February 28, 2023, as "Rare Disease Day". One of the most popular sessions at the 2022 #NORDSummit was, Mental Health & #RareCancers. This session allowed attendees the opportunity to gain insights on supporting patients & families struggling with mental health issues. 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. 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. Even after an accurate diagnosis, treatment often is not available because fewer than 500 rare diseases have FDA-approved treatments. WORLDSymposium and the Lysosomal Disease Network (LDN) are separate and independent entities and are not affiliated in any way. http://ow.ly/5Wzs50Lj6CJ #NORDSummit. Join the National Organization for Rare Disorders (NORD) October 15-17, 2023 for the Rare Diseases and Orphan Products Breakthrough Summit. A global community in the heart of London. 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. WebKatie Wise - EveryLife Foundation for Rare Diseases | Rare Disease Conference 2023 - Rare Disease Innovation & Partnership Summit Speaker Agenda Speakers Sponsors & Exhibitors Contact Katie Wise Young Adult Rare Representative at EveryLife Foundation for Rare Diseases Profile Kaitlyn (Kate) Wise grew up in Westchester, NY. Download the presentation, Antonis KATTAMIS, Professor of Pediatric Hematology-Oncology, President, Hellenic Society of Pediatric Hematology-Oncology Sessions will include information from patients, physicians, and researchers regarding wAIHA clinical trials, current treatments, access to care, federal and state advocacy efforts, etc. If you would like to find out more about how we manage your personal information please see our privacy policy. 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? Sheraton Harbor Island, 1380 Harbor Island Dr, San Diego, CA 92101. Connect, exchange with clinicians and health policy makers. 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. Crickresearchers are working at the forefront of the scientific response to answer some of the most urgent questions about the SARS-CoV-2 pathogen, from how we can improve testing, to why its deadly in some people but causes no symptoms in others. The site is secure. Thank you for the well-organized machinery that allowed us opportunities to arrange meetings. July 1, 2022Abstract submission website open. 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. , Continue reading "Skate Under the Stars: A Rare Disease Celebration". WebMarketsandMarkets Infectious Disease and Molecular Diagnostics Conference 22nd - 23rd June 2023 Boston, USA The Diagnostics sector has emerged as a prominent game changer in healthcare, because of many technological advancements taking Patients must rely on the personal and individualized medical advice of their qualified health care professionals before seeking any information related to their particular diagnosis, cure or treatment of a condition or disorder. WebGT+R 2023 is a novel event, curating seasoned experts from across the gene therapy and rare disease therapeutic research and development industries. 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. The Francis Crick Institute1 Midland RoadLondon NW1 1AT, View upcoming changes to normal opening times. Rare Disease Day at NIH 2023 Tuesday, February 28, 2023, 9:00am to 5:00pm (registration required) NIH Natcher Conference Center (Building 45) Rare Disease Day takes place worldwide, typically on or near the last day of February each year, to raise awareness among policymakers and the public about rare diseases and their Download the presentation, Panagiota MITROU, Deputy Head of the Autonomous Department of Therapeutic Protocols & Patient Registries, Ministry of Health Engage with the FDA to provide your perspectives as a patient, caregiver or family member. Learn more about how you can attend this event or add it to your calendar. Presentations. Washington, DC 20036 Please enable JavaScript in your web browser to get the best experience. 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. Click here to access the Guide for Authors or to Submit Your Paper. 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. Join our mailing lists to receive updates about our latest research and to hear about our free public events and exhibitions. On October 17 and 18, the National Organization for Rare Disorders (NORD) hosted the Rare Diseases and Orphan Products Breakthrough Summit. Join us virtually or in-person on March 10 for the 10th Annual Rare Disease Symposium. By submitting, you agree to receive email communications from Terrapinn, including upcoming promotions and discounted tickets and news. About Rare Disease Day . ContactOOPDOrphanEvents@fda.hhs.gov | 301-796-7634 February 21, 2023: Robert J. Gorlin Symposium and Emerging Trends February 22-26, 2023: Research Meeting Hilton Orlando, Orlando, FL, USA Scientific Broadly acclaimed speakers, the most recent frameworks, methodologies, and the most current updates in this field are indications of this conference. Rare Disease Conferences 2023/2024/2025 lists relevant events for national/international researchers, scientists, scholars, professionals, engineers, exhibitors, sponsors, academic, scientific and university practitioners to attend and present their research activities. How are you raising awareness for the rare community this Rare Disease Day? 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. Thank you to all the folks at NORD for your remarkable efforts to make this such a success.. (February 28, 2023) About Rare Disease Day Launched by EURORDIS-Rare Each year, WORLDSymposium hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. All Info for H.Res.181 - 118th Congress (2023-2024): Expressing support for the designation of February 28, 2023, as "Rare Disease Day". 08:00 AM 05:00 PM UTC-12:00. The World Orphan Drug Congress USA is a place where innovation and expertise is showcased, solutions are found and learning is done. 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. WebWorld Rare Disease Day Conference 2023. Working together to build sustainable events, Multiple Myeloma Research Foundation (MMRF), anti-money laundering & financial crime policy. Read full announcement here. WORLDSymposium is an annual research conference dedicated to lysosomal diseases. Vice President, Discovery and Translational Research. Bring together a broad audience including patients, patient advocates, caregivers, health care providers, researchers, trainees, students, industry representatives and government staff. Our primary method for achieving this is by creating exclusive business conferences that gather together the world's smartest thinkers and doers. Google Calendar. Presentation will cover Jaguar and Napo Therapeutics' focus on development and commercialization of 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. All Info for H.Res.181 - 118th Congress (2023-2024): Expressing support for the designation of February 28, 2023, as "Rare Disease Day".