At FDA Rare Disease Day 2026, leaders highlighted new regulatory pathways, faster review programs, and patient-centered innovations accelerating treatments for rare diseases, including NF1 and pediatric cancers. From the Plausible Mechanism Framework to expanded real-world evidence use, the ... At FDA Rare Disease Day 2026, leaders highlighted new regulatory pathways, faster review programs, and patient-centered innovations accelerating treatments for rare diseases, including NF1 and pediatric cancers. From the Plausible Mechanism Framework to expanded real-world evidence use, the message was clear: Urgency, flexibility, and patient voice are driving rare disease drug development forward. The example illustrates how flexible designs and meaningful endpoints can bring treatments to patients with conditions that historically had few options. For many families affected by NF1 and other rare conditions, Rare Disease Day is personal. In my recent LinkedIn newsletter reflecting on Rare Disease Month and my mother’s experience with NF1, I wrote about the intersection of science, advocacy, and gratitude. On Rare Disease Day, the reminder for IPC leaders is clear: rare does not mean irrelevant. Preparedness, surveillance, and stewardship remain foundational. Rare Disease Day 2026 at the Food and Drug Administration carried a tone of urgency, momentum, and collaboration. The theme is ““Moving forward. Looking ahead. An event for patients.” Throughout the event, agency leaders, clinicians, patient advocates, and scientists emphasized that progress in rare diseases depends not only on regulatory flexibility, but on listening carefully to the lived experiences of patients and families.

Original title: “FDA Rare Disease Day 2026: Moving Forward With Urgency, Innovation, and Patient Voice | Infection Control Today”

The Florida Institute for Pediatric Rare Diseases and the Florida State University College of Medicine will recognize Rare Disease Day this week with a The Florida Institute for Pediatric Rare Diseases and the Florida State University College of Medicine will recognize Rare Disease Day this week with a symposium and panel discussion, featuring experts in genomics and its clinical application. Observed annually on the last day in February, Rare Disease Day is a global movement raising awareness for the estimated 300 million people diagnosed with one of more than 6,000 identified rare diseases. This marks the third Rare Disease Day event at the College of Medicine since Florida Gov. Home / News / Health & Medicine / Florida Institute for Pediatric Rare Diseases will host Rare Disease Day symposium and panel discussion

Original title: “Florida Institute for Pediatric Rare Diseases will host Rare Disease Day symposium and panel discussion - Florida State University News”

NORD Calls on Nation to “Show Your Stripes” on Global Rare Disease Day, Feb. 28 NORWELL, Mass., FEB. 23, 2026 – The National Organization for Rare Disorders (NORD®) is calling attention to rare diseases … Continue reading "More Than 30 Million Americans Living with Rare Diseases"

Original title: “More Than 30 Million Americans Living with Rare Diseases”

CHICAGO, Feb. 23, 2026 (GLOBE NEWSWIRE) -- The Foundation for Sarcoidosis Research (FSR) has been named a 2025 RareVoice Awards recipient by EveryLife Foundation for Rare Diseases, earning national recognition for Federal Advocacy by a Patient or Organization. Foundation for Sarcoidosis Research is proud to receive 2025 RareVoice Award for Federal Advocacy and attend Rare Disease Month on Capitol Hill... Awardees are selected from nominations submitted by patients, caregivers, advocates, and organizations across the rare disease community. “Advocacy means standing in the gap until systems change and patients are seen, heard, and served”, said Mary McGowan, President and CEO of FSR. As part of these efforts, Rare Disease Week on Capitol Hill will take place the last week of February, bringing together patients, caregivers, advocates, researchers, and policymakers from across the country. The 2025 RareVoice Awardees will be featured during Rare Disease Week on Capitol Hill, including at the RareVoice Success Panel, where awardees will share their advocacy journeys and policy impact. sarcoidosis rare disease february chronic illness award rare disease month advocacy fmla capitol hill GlobeNewswire · January 21, 2026 11:08 ET | Source: Foundation for Sarcoidosis Research

Original title: “Foundation for Sarcoidosis Research Receives 2025 RareVoice Award for Federal Advocacy During Rare Disease Month”

CHICAGO, Feb. 23, 2026 (GLOBE NEWSWIRE) -- The Foundation for Sarcoidosis Research (FSR) has been named a 2025 RareVoice Awards recipient by EveryLife Foundation for Rare Diseases, earning national recognition for Federal Advocacy by a Patient or Organization. Foundation for Sarcoidosis Research is proud to receive 2025 RareVoice Award for Federal Advocacy and attend Rare Disease Month on Capitol Hill... Awardees are selected from nominations submitted by patients, caregivers, advocates, and organizations across the rare disease community. “Advocacy means standing in the gap until systems change and patients are seen, heard, and served”, said Mary McGowan, President and CEO of FSR. As part of these efforts, Rare Disease Week on Capitol Hill will take place the last week of February, bringing together patients, caregivers, advocates, researchers, and policymakers from across the country. The 2025 RareVoice Awardees will be featured during Rare Disease Week on Capitol Hill, including at the RareVoice Success Panel, where awardees will share their advocacy journeys and policy impact. sarcoidosis rare disease february chronic illness award rare disease month advocacy fmla capitol hill GlobeNewswire · January 21, 2026 11:08 ET | Source: Foundation for Sarcoidosis Research

Original title: “Foundation for Sarcoidosis Research Receives 2025 RareVoice Award for Federal Advocacy During Rare Disease Month”

Nationwide initiative encourages health-care providers to boost rare-disease knowledge and early diagnosis   BOSTON and NORWELL, Mass., FEB 23, 2026 – In recognition of Rare Disease Day, the National Organization for Rare Disorders (NORD®) … Continue reading "Earn Your Stripes: NORD Launches Rare Disease Day® CME Challenge to Close Critical Gaps in Rare Disease Education"

Original title: “Earn Your Stripes: NORD Launches Rare Disease Day® CME Challenge to Close Critical Gaps in Rare Disease Education”

In Catalysts in Action: How Patient Groups Spark Drug Development (March 11, 12:05-1 p.m. ET), leaders from the RTW Foundation, Elly’s Team, and the TESS Research Foundation will share how advocacy organizations are not only raising awareness but building research programs, funding science, and creating pathways to treatment where none previously existed. As Rare Disease ... In Catalysts in Action: How Patient Groups Spark Drug Development (March 11, 12:05-1 p.m. ET), leaders from the RTW Foundation, Elly’s Team, and the TESS Research Foundation will share how advocacy organizations are not only raising awareness but building research programs, funding science, and creating pathways to treatment where none previously existed. As Rare Disease Day and the Empowering Patients Summit approach, it is worth reflecting on what true progress requires: collaboration, persistence, and listening to the voices whose lived expertise helps guide researchers, industry, and research professionals to continue working towards greater hope. Each year, on the last day of February, the rare disease community and numerous stakeholders unite to recognize Rare Disease Day. This spirit will be… The day serves as a global movement to raise awareness, advance research, and amplify the voices of the 300 million people worldwide affected by rare diseases. With more than 10,000 known rare diseases and 95% lacking an FDA-approved treatment, the need for collaboration, advocacy, and scientific innovation has never been clearer. Since the passage of the Orphan Drug Act in 1983, more than 1,300 orphan drugs have been approved, yet thousands of rare diseases still lack approved treatments, with too many families still waiting. At ASGCT, advancing cell and gene therapies (CGTs) means more than advancing science. It means centering the people who stand to benefit from it. Patients and caregivers have not been passive participants in this progress but have taken the driver's seat by advocating for research, funding early programs, and not letting off the gas when a roadblock gets in the way.

Original title: “Centering Patient Voices: Rare Disease Day and Empowering Patients…”

International Rare Disease Day was marked on 22nd February 2026 in Lucknow by the Rare Diseases India Foundation and SGPGI. Hundreds participated in various activities, including an awareness walk, discussions, and cultural performances. The event highlighted challenges and pushed for improved ... International Rare Disease Day was marked on 22nd February 2026 in Lucknow by the Rare Diseases India Foundation and SGPGI. Hundreds participated in various activities, including an awareness walk, discussions, and cultural performances. The event highlighted challenges and pushed for improved care and policy enhancements for rare disease patients. International Rare Disease Day was commemorated on 22nd February 2026 in a major event at Lucknow, organized collaboratively by the Rare Diseases India Foundation and the Sanjay Gandhi Postgraduate Institute of Medical Sciences. The event drew significant attention with hundreds of rare disease patients, medical professionals, and families engaging in activities designed to highlight the unique challenges of rare disease communities. Key concerns about policy inadequacies, especially funding caps under the National Policy for Rare Diseases 2021, were voiced by Mr. Saurabh Singh of RDIF, emphasizing the need for policy revisions and sustainable health solutions. The day concluded with a call for unity and ongoing advocacy to support the rare disease community effectively.

Original title: “Uniting Voices: Rare Disease Advocacy Day in Lucknow | Health”

New Delhi [India], February 23: International Rare Disease Day was observed with great commitment and solidarity on 22nd February 2026 through a collaborative initiative of Rare Diseases India Foundation (RDIF) and Sanjay Gandhi Postgraduate Institute of Medical Sciences (SGPGI), Lucknow. New Delhi [India], February 23: International Rare Disease Day was observed with great commitment and solidarity on 22nd February 2026 through a collaborative initiative of Rare Diseases India Foundation (RDIF) and Sanjay Gandhi Postgraduate Institute of Medical Sciences (SGPGI), Lucknow. The event witnessed the participation of hundreds of rare disease patients and their families, along with leading doctors, pediatric specialists, clinicians, physiotherapists, and healthcare professionals. The program also included cultural performances by patients and families, celebrating resilience and hope within the rare disease community. Dr. Devendra Gupta, Chief Medical Superintendent of SGPGI, underlined the critical role of Patient Advocacy Groups in bridging the gap between patients, healthcare providers, and policymakers. The Program concluded with a strong message of unity, awareness, and collective responsibility -- reaffirming the commitment to build an inclusive healthcare ecosystem where rare disease patients are not invisible but empowered. (ADVERTORIAL DISCLAIMER: The above press release has been provided by NewsVoir. ANI will not be responsible in any way for the content of the same.) (This content is sourced from a syndicated feed and is published as received. The Tribune assumes no responsibility or liability for its accuracy, completeness, or content.) ... Advertorial DisclaimerhealthcareNewsVoirPatient advocacyPediatric specialistsPhysiotherapyRare disease day Kaushik Mandal, Head of the Department of Pediatrics, SGPGI Lucknow, assured the community that the medical fraternity stands firmly with rare disease patients. He stated, "We are available 24x7 to support this community because we understand their suffering very closely." Mr. Saurabh Singh, Co-Founder & Director of Rare Diseases India Foundation (RDIF), stated that the Rs.

Original title: “International Rare Disease Day 2026 Observed on 22nd February 2026 at SGPGI, Lucknow - The Tribune”

Living with a rare disease means navigating stigma, isolation, and uncertainty. For young people, these challenges are compounded by the pressures of growing up, starting… Continue reading Raising Youth Voices 2026: Shaping the Future of the Rare Disease Community. Over fifty young people applied, and while the final selection was difficult, the following advocates were selected to represent their regions: Liam (North America – USA), Maksym (Europe – Ukraine), Su (Asia – China), Pablo (South America – Colombia), Vincent (Africa – Zimbabwe) and Stephanie (Oceania – Australia). Each representative was chosen based on their leadership, advocacy experience, communication skills, and commitment to advancing the rare disease agenda through innovative, local Rare Disease Day projects. With over 90 registrations, the event drew a diverse audience consisting of medical students, patient organisation leaders, researchers, and industry representatives. The panel, moderated by Global Rare Disease Day Lead Rhiannon Walls, started with presentations and went into a discussion that highlighted both the shared issues and the strength found in the diversity of people’s approaches to tackling these issues. Liam (21) spoke about the “stigma around sharing your rare disease” in public forums. Stephanie (30) shared of finding a balance between sharing her daughter Mila’s diagnosis with infantile neuroaxonal dystrophy (INAD) in their advocacy and her right to privacy, describing the constant pressure to share things beyond the boundaries of what her family would be comfortable with. Both she and Liam also stressed the importance of showing through social media that people are more than their rare disease. Mila is “a person, and she has a right to dignity”. While social media is crucial to the lives of youth today, acting as a form of democratisation in which everyone has the chance to have a voice, it also comes with risks and challenges, Liam admits, “the internet can be a cruel place”. Liam went on to discuss the strengths of the healthcare and advocacy infrastructure in the United States, while reminding the global audience how fragile access to care and treatments can be without health insurance.

Original title: “Raising Youth Voices 2026: Shaping the Future of the Rare Disease Community. - Rare Disease Day 2026”

Prions are misfolded proteins in the body that cause fatal illnesses in people and animals.

Original title: “About Prion Diseases”

Find a list of fungal diseases and navigate to their sites for more information.

Original title: “Types of Fungal Diseases”

Angela is the Founder and President ... disease patients in California. Cal Rare started the California Rare Disease Legislative Caucus that now includes 25% of the State Legislature. In 2018, Angela was the recipient of the Rare Voice Award for State Advocacy from the Rare Disease Legislative Advocates. In 2019, she was named a Top 100 Social Media Advocate for Rare Disease and in 2020, a Top 50 Rare Disease Awareness Advocate by the International Pain Foundation... Angela is the Founder and President of California Action Link for Rare Diseases (Cal Rare), a non-profit membership coalition of rare disease stakeholders that began in 2017 and is dedicated to improving the lives of rare disease patients in California. Cal Rare started the California Rare Disease Legislative Caucus that now includes 25% of the State Legislature. In 2018, Angela was the recipient of the Rare Voice Award for State Advocacy from the Rare Disease Legislative Advocates. In 2019, she was named a Top 100 Social Media Advocate for Rare Disease and in 2020, a Top 50 Rare Disease Awareness Advocate by the International Pain Foundation. Shannah Hudson is a rare disease and newborn screening advocate, and mom to Emmalyn who has Glutaric Aciduria/Acidemia Type 1 (GA-1). She founded a registered 501(c)(3) nonprofit organization, Mississippi Metabolics Foundation (MMF), to raise awareness, educate, support, and advocate for patients in Mississippi with rare diseases, specifically Inborn Errors of Metabolism. She currently serves as the CEO and board President for MMF and is a member of the EveryLife Foundation’s Community Congress and a founding member organization with the Global Genes Global Advocacy Alliance (GAA). She is on the Patient Experience Bureau for the Child Neurology Foundation, the Patient/Consumer Representative for Mississippi for the Southeast Regional Genetics Network (SERN), and a Rare Genetic Disease Patient Ambassador with Illumina, Inc. Shannah also serves in advocacy efforts with various other patient organizations including the National Organization for Rare Disorders (NORD), NORD’s MS Rare Action Network (RAN), Genetic Alliance, Baby’s First Test, and the Organic Acidemia Association (OAA). Because of the rarity of this disorder and having it 15 years before a diagnostic test existed, I incurred severe and multiple disabilities including blindness and paralysis. I earned a BA in Mathematics in 2001 from Rhodes College in Memphis, TN. I serve on the Patient Advocacy Council for the Guthy Jackson Charitable Foundation. I am a co-group leader for the Oklahoma Support Group of the Siegel Rare Neuroimmune Association.

Original title: “RDW 2026 - EveryLife Foundation for Rare Diseases”

Dr. Lauren Jansen, an assistant professor in the Department of Family Medicine at the University of Massachusetts Chan Medical School, and Brianna Loeck, and epidemiologist with the state of Nebraska, discuss an outbreak of campylobacteriosis that occurred in Nebraska in 2021.

Original title: “Campylobacteriosis Outbreak Linked to Municipal Water, Nebraska, USA, 2021”

This report describes regional increases in Valley fever in Arizona during 2005-2022.

Original title: “Regional Increases in Incidence of Coccidioidomycosis (Valley Fever) — Arizona, 2005–2022”

With Rare Disease Day approaching on Feb. 28, the Florida Institute for Pediatric Rare Diseases at the Florida State University College of Medicine is Support from the Sunshine Genetics Act has enabled us to build one of the nation’s most forward-looking pediatric genomics programs. We also collaborate closely with universities, hospitals, foundations, legislators, and advocacy organizations to connect families with clinical trials and support networks. This coordination ensures that families are not navigating rare disease care alone. Each diagnosis also strengthens the scientific foundation needed to develop new therapies and, ultimately, preventive strategies. At Florida State University, the Florida Institute for Pediatric Rare Diseases and the Sunshine Genetics Pilot Program are working together to deliver these benefits statewide. The numbers are eye-opening: There are more than 7,000 pediatric rare diseases affecting approximately 30 million people in the United States, including 15 million children. The Sunshine Genetics Act was championed by Rep. Adam Anderson (R-Palm Harbor), whose son Andrew passed away from Tay-Sachs disease at just 4 years old and inspired his push to create meaningful legislation. Pradeep Bhide, director of the Florida Institute for Pediatric Rare Diseases, views Rare Disease Day as a reminder of research’s potential to improve the quality of life for children and families.

Original title: “Rare Disease Day 2026: Medical expert available for interviews on the State of Florida's optimistic outlook - Florida State University News”

Families and advocates will gather Feb. 28 to spotlight rare diseases and connect those impacted in Southern Arizona. TUCSON, Ariz. (KGUN) — Rare diseases affect fewer than 200,000 Americans, and families in Tucson will gather Feb. The Rare Disease Day event will be held next Saturday at the Children’s Museum Tucson, bringing together families and organizations that support people living with rare conditions. According to the U.S. Department of Health and Human Services, doctors still do not know exactly what causes many rare diseases. While autism itself is not classified as a rare disease, Miller said there is significant overlap within the community.

Original title: “Tucsonans unite to raise awareness for Rare Disease Day”

Rare Disease Day inspires solidarity and pushes for greater equity in healthcare systems around the world. Through storytelling platforms, social media engagement, webinars, and community driven activities, Rare Disease Day inspires solidarity and pushes for greater equity in healthcare systems around the world. The 2026 global Rare Disease Day campaign continues to emphasize community participation and awareness building. With a growing library of digital resources, webinars, patient stories, and advocacy tools, the campaign provides individuals, families, clinicians, and organizations with the means to get involved. Rare Disease Day, observed annually on the last day of February, continues to unite millions around the world in advocating for fair access to diagnosis, treatment and support for people living with rare conditions. Taking place on Thursday, 5 March 2026, this free online event, hosted by Genetic Alliance UK, welcomes participants from across all four UK nations and beyond. The programme includes insights from individuals living with rare diseases, contributions from leading policy makers, and discussions centred on improving healthcare pathways, support networks, and diagnosis experiences.

Original title: “Celebrating Rare Disease Day 2026 | DEBRA UK”

He is also a former EveryLife Foundation Board Chair, ending his most recent term at the end of 2025. Lifetime Achievement awardees receive an "Abbey" statuette commissioned specially for the RareVoice Awards. In 2025, more than 800 advocates from 49 states and representing over 184 patient organizations attended over 363 meetings with Members of Congress and/or staffers during Rare Disease Week. Shannon von Felden, Vice President of Advocacy ... He is also a former EveryLife Foundation Board Chair, ending his most recent term at the end of 2025. Lifetime Achievement awardees receive an "Abbey" statuette commissioned specially for the RareVoice Awards. In 2025, more than 800 advocates from 49 states and representing over 184 patient organizations attended over 363 meetings with Members of Congress and/or staffers during Rare Disease Week. Shannon von Felden, Vice President of Advocacy at the EveryLife Foundation and head of RDLA, believes that the accessibility of the event helps the organization to see continued growth year after year: "There is no charge for advocates to participate in Rare Disease Week, and advocacy training is provided for all who attend, reinforcing the commitment of RDLA and the EveryLife Foundation to ensuring access and inclusion for the entire rare disease community. 18, 2026 /PRNewswire/ -- For the 15th consecutive year, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) will welcome advocates from 49 states, the District of Columbia, and Puerto Rico to Rare Disease Week on Capitol Hill, February 24 through February 26, 2026, in Washington, D.C. A free event, Rare Disease Week convenes patients, caregivers, advocates, and policymakers to elevate the rare disease patient voice and advance policies that improve the lives of millions of Americans living with rare conditions. As part of the EveryLife Foundation's ongoing efforts to continually enhance the event — and informed directly by attendee feedback — Rare Disease Week 2026 will introduce several important updates designed to strengthen community connection, expand storytelling opportunities, and create a more intentional advocacy experience. The conference will also feature a gallery showcasing the work of the 2025 Rare Artists, highlighting the intersection of art, advocacy, and lived experience within the rare disease community. On Thursday, February 26, the Senate Special Committee on Aging will hold a hearing at 9:30 a.m. ET to examine the FDA's role in rare disease therapy development. Then at 12:30 p.m. ET attendees will have the opportunity to attend the Rare Disease Congressional Caucus Briefing before meeting with their Members of Congress on Capitol Hill. The EveryLife Foundation's Rare Disease Week activities end with a closing reception.

Original title: “The EveryLife Foundation for Rare Diseases Marks 15th Annual Rare Disease Week on Capitol Hill”

Through global initiatives, ... the Foundation brings visibility to topics that are often unseen or misunderstood, ensuring that the real experiences of rare disease communities are recognised. ... Rare Disease Day is a powerful reminder that progress does not happen in isolation. It depends on collaboration between patients and families, advocacy groups, healthcare ... Through global initiatives, publications and partnerships, the Foundation brings visibility to topics that are often unseen or misunderstood, ensuring that the real experiences of rare disease communities are recognised. ... Rare Disease Day is a powerful reminder that progress does not happen in isolation. It depends on collaboration between patients and families, advocacy groups, healthcare professionals, researchers and scientists, policymakers, the pharmaceutical industry. Rare diseases are more common than most people imagine, in how many lives they touch and in how profoundly they Advocacy engagements, collaborating with global patient organisations · Co-created resources, designed with and for patients, families and caregivers · We also share what we learn in our own awareness activities, ensuring that the voices of patients reach wider audiences. This is why Rare Disease Day is a central moment in the Ipsen calendar: it allows us to stand alongside the global rare disease community to raise understanding and amplify impact. Fondation Ipsen is a non-profit foundation, operating under the aegis of the Fondation de France, and dedicated to raising awareness and improving the lives of people living with rare diseases.

Original title: “Rare Disease Day 2026: Listening to Patients to Imagine Better Futures - Global”