Come to SouthPark Mall (Atrium near Lego Store) on 2/28 from 10-2 to celebrate Rare Disease Day with friends from the Ward Foundation. Activities, zebra photo

Original title: “Rare Disease Day at SouthPark Mall - National Organization for Rare Disorders”

The “Omirge Sen” Foundation was established to support families whose members suffer from hereditary neuromuscular diseases, including myopathies (Duchenne/Becker), spinal muscular atrophy (SMA), congenital myasthenia,… Continue reading Stories of Our Little Heroes The “Omirge Sen” Foundation was established to support families whose members suffer from hereditary neuromuscular diseases, including myopathies (Duchenne/Becker), spinal muscular atrophy (SMA), congenital myasthenia, Charcot-Marie syndrome, and others. This video was filmed featuring our beneficiaries for Rare Disease Day to raise awareness of Duchenne muscular dystrophy.

Original title: “Stories of Our Little Heroes - Rare Disease Day 2026”

The “Omirge Sen” Foundation was established to support families whose members suffer from hereditary neuromuscular diseases, including myopathies (Duchenne/Becker), spinal muscular atrophy (SMA), congenital myasthenia,… Continue reading Story of our little heroes The “Omirge Sen” Foundation was established to support families whose members suffer from hereditary neuromuscular diseases, including myopathies (Duchenne/Becker), spinal muscular atrophy (SMA), congenital myasthenia, Charcot-Marie syndrome, and others. This video was filmed featuring our beneficiaries for Rare Disease Day to raise awareness of Duchenne muscular dystrophy.

Original title: “Story of our little heroes - Rare Disease Day 2026”

Enhanced Genomics has appointed Katerina Leftheris to its board of directors

Original title: “Katerina Leftheris”

ASrid is a non-profit and sole organization for multi-stakeholders in Rare and Intractable diseases. ASrid is committed to providing the valuable service/system for “connecting” and… Continue reading Advocacy Service for Rare and Intractable Diseases ASrid is a non-profit and sole organization for multi-stakeholders in Rare and Intractable diseases. ASrid is committed to providing the valuable service/system for “connecting” and “creating” with/among them. ... For patients and with patients, always.

Original title: “Advocacy Service for Rare and Intractable Diseases - Rare Disease Day 2026”

Infographic about how the National Diabetes Prevention Program reduces the risk of type 2 diabetes.

Original title: “National Diabetes Prevention Program Infographic”

Learn about the statistics, health risks, and prevention of prediabetes.

Original title: “Prediabetes: Could It Be You?”

The NORD Students for Rare chapter at Quinnipiac University, in collaboration with the Center for Interprofessional Education and Connecticut Children’s The NORD Students for Rare chapter at Quinnipiac University, in collaboration with the Center for Interprofessional Education and Connecticut Children’s Research Institute, are hosting “Rare Disease Day 2026: More Than You Can Imagine!”. Join us on February 20, 2026, as a team of patients, scientists, administrators, and advocates show how more research, more breakthroughs, and more action are bringing more hope to the Osteogenesis Imperfecta rare disease community. Your gift today fuels progress in the lab, in clinics, on Capitol Hill and for rare families who can’t wait another year for answers.

Original title: “Quinnipiac Rare Disease Day 2026: More Than You Can Imagine - National Organization for Rare Disorders”

For families facing ultrarare diseases — conditions affecting perhaps dozens of people globally — the path can be even more daunting. There may be no research, no clinical trials, no pharmaceutical company interested in developing a therapy. So, today’s rare disease patients and patient ... For families facing ultrarare diseases — conditions affecting perhaps dozens of people globally — the path can be even more daunting. There may be no research, no clinical trials, no pharmaceutical company interested in developing a therapy. So, today’s rare disease patients and patient organizations are jumping in to fund research, run natural history studies, hire scientists, and in some cases, launch biotech companies. In the U.S., 1 in 10 individuals are affected by rare diseases, yet our healthcare system still isn’t built to serve rare disease patients. When an answer finally comes, families face another sobering reality: 95% of rare diseases have no approved treatment. Families are left to navigate a fragmented system, often paying out-of-pocket for specialists, genetic testing, or experimental care. ... These are not just medical challenges; they are human ones. Patients become advocates, testifying before regulators and fundraising for clinical trials. Behind many breakthroughs in modern medicine are lessons first learned in rare disease science. Gene therapy, personalized medicine, patient-driven research — these innovations emerged from the rare disease community’s refusal to accept “nothing can be done.”

Original title: “From Diagnosis to Treatment: How Rare Disease Families Navigate the Path to Genetic Therapy”

Compact, hands-on CME on gene & cell therapies for rare diseases—a concise introduction for primary care providers. Gene and cell therapy is no longer medicine of the future—it has become a clinical reality. For general practitioners, foundational knowledge in this area is essential. This is where the new course series "Rare Diseases in Practice" comes in. Gene- and cell-based therapies are rapidly evolving from experimental concepts to clinical reality. Especially in the field of rare diseases, these approaches are opening up causal treatment options for the first time, where previously only symptomatic therapies were available. The live talk is a joint event by the Eva Luise and Horst Köhler Foundation, the National Strategy for Gene and Cell-Based Therapy, and the German Society for Gene Therapy e.V. ... Save the date: The live talk on October 13, 2025, will take place from 6:00 to 7:30 p.m. as an interactive live webinar on the STREAMED UP video platform. On… ... Neuronal ceroid lipofuscinoses (NCL) are the most common types of neurodegenerative diseases in childhood and adolescence. NCL specialist Dr. med. Angela Schulz from UKE Hamburg-Eppendorf provides an overview of the… ... On Rare Disease Day, February 28, 2025, our live and interactive training series for outpatient physicians will begin. Annette Grüters-Kieslich, Chair of the Executive Board of the Eva Luise and Horst Köhler Foundation, three distinguished experts presented and explained the current state of these therapies on February 24, 2026, in a live talk on the STREAMED UP platform: Prof. Dr. Hildegard Büning, Hannover Medical School: “Gene- and cell-based therapies – when concepts become clinical reality”

Original title: “Gene & Cell Therapy for Rare Diseases | CME Compact”

JANESVILLE – Rare Disease Day is on the minds again of Janesville Craig's Global Scholars student group. Sign up for news in your inbox every day! ... Members of the Craig Global Scholars shown on the Blain Gilbertson Family Heritage Bridge in downtown Janesville, are (from left) freshmen Kelsey Goodger, Lauren Groessel and Alyssa Nelson; sophomore Andrew Hopkins; junior Marlon Kohn; senior Ella Shea; and sophomores Bella Trickie and Kayana Serrano. ... JANESVILLE — Rare Disease Day is on the minds again of Janesville Craig’s Global Scholars student group. The group also plans traditional means of promoting Rare Disease Day, including “just talking a little bit about why we’re doing this service project and what we’re doing and just Rare Disease Day in general because a lot of people don’t know about it,” Shea said. As in past years, the group is once again working on big projects to bring awareness to the 2026 Rare Disease Day, which is Feb. Last year, Gov. Evers declared Feb. 28, 2025, as Rare Disease Day in Wisconsin.

Original title: “'We have a lot of people backing us up.' Craig Global Scholars again raising awareness of Rare Disease Day | Local News | gazettextra.com”

Powerful, authentic Rare Disease Day quotes to inspire awareness, foster empathy, and ignite hope—curated with care for patients, families, clinicians, and advocates. This article curates and contextualizes 22 carefully selected Rare Disease Day quotes—not just for sharing on social media, but for grounding advocacy in authenticity, science, and humanity. Rare disease journeys often begin in silence: unanswered questions at pediatric check-ups, dismissed symptoms labeled “anxiety” or “growing pains,” stacks of inconclusive test results. By the time many receive a confirmed diagnosis—averaging 4.8 years in the U.S., per the National Organization for Rare Disorders (NORD)—patients have already internalized doubt, invisibility, and exhaustion. Tip: When selecting or sharing a Rare Disease Day quote, ask: Does it center the lived experience—not just the condition? Does it honor complexity without romanticizing struggle? If it passes both tests, it’s ready to resonate. These quotes were sourced from patients, caregivers, researchers, clinicians, and global advocacy leaders—including statements delivered at WHO forums, published in peer-reviewed journals, and shared during Rare Disease Day vigils across 100+ countries. Cross-check against primary sources (e.g., official Rare Disease Day press kits, GARD publications, or verified speaker transcripts). In clinical settings: Print short quotes on adhesive notes and place them inside patient chart folders—not as platitudes, but as cognitive cues for providers. Example: Place Dr. Tanaka’s quote next to the “History of Present Illness” section. In research proposals: Use advocacy-aligned quotes in the “Significance” section to humanize statistical aims. “serious”), but by functional purpose: validation, advocacy, clinical partnership, scientific resolve, and intergenerational wisdom. Notice how none rely on metaphors of “battling” or “winning.” That’s intentional. The Global Alliance of Rare Disease Organizations (GARD) explicitly discourages militaristic language in its 2023 Communications Guidelines, citing evidence that such framing increases patient guilt when progression occurs and undermines holistic care goals.

Original title: “Rare Disease Day Quotes To Inspire Awareness & Hope”

Pseudo-Outbreak of Purpureocillium lilacinum Skin Infections at a Dermatology Clinic — Washington, 2024

Original title: “Pseudo-Outbreak of Purpureocillium lilacinum Skin Infections at a Dermatology Clinic — Washington, 2024”

The oral version of Wegovy has made GLP-1 medicines more accessible and acceptable to many patients who had steered clear of injectable versions, healthcare analytics firm Truveta suggests.

Original title: “Oral Wegovy Brings in Thousands of New GLP-1 Patients”

Biopharma professionals need to understand today’s job market and how they can stand out to position themselves for success. Three talent acquisition and recruiting experts discussed these topics in a BioSpace webinar, from the importance of contract work to the value of an advocate.

Original title: “7 Key Insights Into the 2026 Job Market and Hiring Landscape”

Learn more about Rare Disease Day 2026 and how you can get involved. Rare diseases can be genetic, infectious or environmental in origin, include allergies, or take the form of rare cancers. Their impact reaches far beyond health, work, support, and care. At the same time, the campaign slogan also carries hope: more solidarity, more advocacy, more hope, and more action than one could imagine, driven by patient organisations and communities making Rare Disease Day meaningful in their own local contexts. On 28 February 2026, people across the globe will once again come together to mark Rare Disease Day. Coordinated by EURORDIS–Rare Diseases Europe and powered by a growing network of National Alliance partners, patient organisations, families and advocates it is a day built from local action and global solidarity, and it continues to expand year after year. In this evolving landscape, rare diseases are a defining test of whether Europe can deliver care that responds to complexity: providing timely diagnosis, connecting patients to specialised expertise, supporting cross-border collaboration and ensuring holistic care that addresses physical, mental and social needs throughout life. The Commission’s Life Sciences Strategy and call for evidence have opened further space for rare-disease priorities, and recent progress on the general pharmaceutical package has created regulatory levers to improve development, access and meaningful patient involvement.

Original title: “Rare Disease Day 2026: More Than You Can Imagine - EURORDIS-Rare Diseases Europe”

Learn more about Rare Disease Day 2026 and how you can get involved. The post Rare Disease Day 2026: More Than You Can Imagine   appeared first on EURORDIS-Rare Diseases Europe.

Original title: “Rare Disease Day 2026: More Than You Can Imagine  ”

Understanding Clinical TrialsExplore ... clinical trials · Access and AffordabilityExplore SCN efforts toward improving accessibility and adoption of advanced therapeutics ... Sponsorship OpportunitiesPromote your organization to top regenerative medicine researchers and companies · AwardsNominate outstanding research for the Till & McCulloch Award ... "For the regenerative medicine community, especially those working in rare diseases, these insights ... Understanding Clinical TrialsExplore frequently asked questions about accessing clinical trials · Access and AffordabilityExplore SCN efforts toward improving accessibility and adoption of advanced therapeutics ... Sponsorship OpportunitiesPromote your organization to top regenerative medicine researchers and companies · AwardsNominate outstanding research for the Till & McCulloch Award ... "For the regenerative medicine community, especially those working in rare diseases, these insights matter deeply. Omar Tounekti from Health Canada opened with a clear overview of how cell and gene therapy trials are reviewed in Canada (much like any other novel biologic drug), then focused on products targeting rare disease populations. While Canada lacks a formal orphan drug designation, there is significant flexibility within existing frameworks to accommodate small patient populations. At Health Canada, Clinical Trial Applications (CTAs) are reviewed within 30 days, with room to justify limited batch or stability data when appropriate. Health Canada’s openness, combined with SCN’s network of researchers, clinicians, and trainees, creates fertile ground for advancing these therapies responsibly and efficiently. SCN will continue to convene these conversations – bringing together international perspectives that help our community prepare for translation and clinical readiness. The path to rare disease treatment is complex, but by working together and learning from global partners, we can make it a little clearer – and bring hope to patients who need it most. Insights from the Network “For the regenerative medicine community, especially those working in rare diseases, these insights matter deeply. They remind us that strategic planning is as critical as scientific discovery. The success of a therapy doesn’t just depend on the science – it depends on navigating regulatory systems that ensure safety, consistency, and patient […]

Original title: “Preparing for Clinical Trials in Rare Disease: Lessons in Global Regulatory Collaboration - Stem Cell Network”

Only 5% of rare diseases have treatments, but approximately half have patient advocacy organizations; these groups have the potential to play major roles in positively influencing research and development of drugs, clinical trials, and regulations by championing funding and awareness efforts, ... Only 5% of rare diseases have treatments, but approximately half have patient advocacy organizations; these groups have the potential to play major roles in positively influencing research and development of drugs, clinical trials, and regulations by championing funding and awareness efforts, forming connections between experts and drug developers, advocating for changes to regulation to expedite research, facilitating patient registries, or other initiatives. The panel was provided with a guidance document used by the Fibrodysplasia Ossificans Progressiva Association (IFOPA), which provided a foundation upon which the panel was asked to build guidelines for the entire rare disease community. Based on the panel’s input, the researchers drafted guidelines, then revised on the basis of further panel comments. A second draft was reviewed by 6 experts from advocacy organizations and industry before being finalized. The resulting document, titled “Guidelines for Interactions Between Patient Advocacy Organizations and Biopharmaceutical Companies,” comprises 4 sections, highlights of which include the following: A rare disease, by definition, affects only a small number of individuals, but together, all of the approximately 7000 known rare diseases affect an estimated 350 million patients worldwide (including approximately 30 million people in the United States). Only 5% of these diseases have treatments, but approximately half have patient advocacy organizations; these groups have the potential to play major roles in positively influencing research and development of drugs, clinical trials, and regulations by championing funding and awareness efforts, forming connections between experts and drug developers, advocating for changes to regulation to expedite research, facilitating patient registries, or other initiatives. paper in Orphanet Journal of Rare Diseases describes efforts to address unmet needs for guidelines on day-to-day decision making by rare disease patient advocacy organizations when working with biopharmaceutical companies.

Original title: “New Guidelines for Rare Disease Advocacy Groups to Navigate Interactions With Pharma | AJMC”

Companies developing therapies for rare diseases frequently decline compassionate use requests not out of indifference, but out of rational business and regulatory concern. These concerns often include potential risk to ongoing or future clinical trials; fear that adverse events could complicate ... Companies developing therapies for rare diseases frequently decline compassionate use requests not out of indifference, but out of rational business and regulatory concern. These concerns often include potential risk to ongoing or future clinical trials; fear that adverse events could complicate regulatory review; limited manufacturing capacity, particularly for biologics and gene therapies; and resource constraints within small or mid-size biotech companies. Even when promising clinical trials exist, and the FDA allows expanded access, rare disease patients like my granddaughter are often left out. Rare-disease drug development depends on public trust: trust from caregivers, clinicians, and advocates who participate in trials, share data, and champion innovation. When families see that investigational therapies exist but are categorically unavailable to those who do not “fit the protocol,” that trust erodes. Yet for many patients — including my granddaughter — access to those therapies remains out of reach because they fall outside clinical trial eligibility criteria such as age or current medications. This is where the system breaks down. The U.S. has a well-established legal pathway for expanded access, often referred to as compassionate use. Contrary to common perception, the FDA is rarely the barrier.

Original title: “My granddaughter has a rare disease. But clinical trials often exclude patients like her”