Leaders across research, clinical care, industry, and patient advocacy convene to accelerate rare disease breakthroughs from discovery to real-world patient impact Norwell, Ma., Feb. 5, 2026 — The National Organization … Continue reading "NIH Director Dr. Jay Bhattacharya to Headline Second Annual NORD Rare Disease Scientific Symposium Focused on Accelerating Innovation"

Original title: “NIH Director Dr. Jay Bhattacharya to Headline Second Annual NORD Rare Disease Scientific Symposium Focused on Accelerating Innovation”

Learn more about our European Regional Task Force on Rare Diseases, and how you can apply to join! The post Join our European Regional Task Force on Rare Diseases appeared first on EURORDIS-Rare Diseases Europe.

Original title: “Join our European Regional Task Force on Rare Diseases”

Redefining the future of customer relationships in biopharma

Original title: “Connect more”

CDC Disease Detective Camp - David J. Sencer CDC Museum

Original title: “CDC Museum Disease Detective Camp”

See where PHAP classes are across the United States.

Original title: “Where Associates Work”

Ahmed Moussa is the new Sanofi Country Lead for UK & Ireland

Original title: “Ahmed Moussa”

Anthrax is a serious bacterial disease found naturally in soil that affects animals and people.

Original title: “About Anthrax”

This has been the reasoning for the withdrawal of market authorization for GlyberaR (alipogene tiparvovec) and Strimvelis as well as the abandonment of scores of other clinical-stage, or near-clinical stage, rare disease CGTs1. The American Society of Gene and Cell Therapy (ASGCT) has recently ... This has been the reasoning for the withdrawal of market authorization for GlyberaR (alipogene tiparvovec) and Strimvelis as well as the abandonment of scores of other clinical-stage, or near-clinical stage, rare disease CGTs1. The American Society of Gene and Cell Therapy (ASGCT) has recently undertaken a series of initiatives designed to maximize access for patients to these life-saving therapies. The first of these was the launching of a task force for access to commercially pre-viable (CPV) CGTs2 consisting of stakeholders from academia, industry, government, and the disease advocacy community. The task force held a series of remote conferences and culminated in an in-person workshop in March 2025 entitled, “Establishing and Maintaining Access to Gene and Cell Therapy for Rare and Ultrarare diseases.” At that workshop, participants identified the high cost and long wait times required for manufacturing and the complex regulatory path as major barriers to accessing CGTs. The rationale for this approach is that the short-term cost of a CGT is more than offset by long-term savings that are realized as the clinical benefit of the therapy lessens the costs for ongoing care by avoiding the need for repeated hospitalizations and emergency department visits (i.e., an expensive curative treatment is more cost-effective than a lifetime of treating the disease symptoms). In addition to these ASGCT initiatives, similar efforts are ongoing among gene therapy professional societies from around the globe, including the European Society of Gene and Cell Therapy, the Japanese As the range of disease treatable by cell and gene therapy (CGT) continues to broaden, the small market size for each rare disease may not enable sufficient return on investment for potential investors.

Original title: “Maximizing Access to Cell and Gene Therapy for Patients with Rare Diseases - Terence R. Flotte, Guangping Gao, 2026”

Awardees are selected by a committee ... by patients, caregivers, advocates, and organizations across the rare disease ecosystem. "Rare disease advocacy is most powerful when it reflects the voices, experiences, and diversity of the community itself," said Shannon von Felden, Vice President of Advocacy at the EveryLife Foundation... Awardees are selected by a committee from nominations submitted by patients, caregivers, advocates, and organizations across the rare disease ecosystem. "Rare disease advocacy is most powerful when it reflects the voices, experiences, and diversity of the community itself," said Shannon von Felden, Vice President of Advocacy at the EveryLife Foundation. 3, 2026 /PRNewswire/ -- The EveryLife Foundation for Rare Diseases (EveryLife Foundation) announced the awardees for the 2025 RareVoice Awards, which honor exceptional advocates who champion and elevate the rare disease patient voice in state and federal policy. The RareVoice Awards recognize individuals and organizations whose advocacy efforts have driven meaningful policy impact on behalf of the rare disease community. The combination of recognition, art, and local representation makes the RareVoice Awards a powerful celebration of advocacy and community. ... Additional RareVoice Awardees will be announced during Rare Disease Week on Capitol Hill, including recipients in the following categories: ... The 2025 RareVoice Awardees will be featured at Rare Disease Week on Capitol Hill, taking place February 24–26, 2026. Learn more about the 2025 RareVoice Awardees. Rare Disease Week on Capitol Hill convenes patients, caregivers, advocates, researchers, and policymakers to highlight the challenges faced by the rare disease community and advance policy solutions that promote innovation, access, and equity. The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization powered by the rare disease community to improve health outcomes by driving change through evidence-based policy, leading science-driven policy and regulatory research, activating the community to advocate for their rights and needs, and strengthening the rare disease community.

Original title: “The EveryLife Foundation for Rare Diseases Announces RareVoice Award Awardees”

Savara’s Early Access Program (EAP) for molgramostim inhalation solution (molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune […] The post Savara’s Early Access Program appeared first on Global Genes.

Original title: “Savara’s Early Access Program”

Rare Disease Day is a global movement to raise awareness and advocate for the more than 300 million people worldwide living with a rare disease. Held annually on the last day of February, this day unites individuals, Over the years, as a special-needs parent navigating the Special Education system for Sophie, Jennifer became interested in learning as much as possible about that process. She began by attending PWSA (USA)’s first Wyatt Special Education Advocacy Training in March of 2013, and went on to complete a rigorous 9-month Special Education Advocacy Training course with the Council of Parent Advocates and Attorneys in May of 2015. She obtained her PhD from Capella University in Education focusing her research on Prader-Willi Syndrome. Dr. McTighe is also a graduate of the William & Mary Law School’s Institute for Special Education Advocacy. Dr. McTighe is currently the chair of the Special Education Advisory Board for the Prader-Willi Syndrome Association-(USA). In addition to Evan’s work with parents, he helped create PWSA (USA)’s Wyatt Special Education Advocacy Training (WSEAT) in 2013, the PWSA (USA) Special Education Advisory Board (SEAB) and created PWSA (USA)’s e-letter “School Times”. Evan’s special education advocacy training includes graduating from the William and Mary College of Law’s Special Education Advocacy Institute and regularly attending the annual Special Education Law Symposium at Lehigh University. It partners with the Southern Poverty Law Center, Protection and Advocacy (P&A) programs, Legal Services Corporations (LSC) and disability organizations on major, systemic disability rights issues involving the Individuals with Disabilities Education Act (IDEA), Americans with Disabilities Act (ADA), and the federal Medicaid Act.

Original title: “Rare Disease Day 2026 - Prader-Willi Syndrome Association | USA”

We will join the Global Chain of ... Jesus in Rare Disease Day colors to honor those impacted and celebrate the resilience of our community. Everyone is invited to join us at the lighting ceremony; no registration is required. Dedicate a Luminary and Request a Grotto Prayer As part of the #LightUpForRare movement, students in the Minor in Science and Patient Advocacy will be creating ... We will join the Global Chain of Lights by illuminating Touchdown Jesus in Rare Disease Day colors to honor those impacted and celebrate the resilience of our community. Everyone is invited to join us at the lighting ceremony; no registration is required. Dedicate a Luminary and Request a Grotto Prayer As part of the #LightUpForRare movement, students in the Minor in Science and Patient Advocacy will be creating visual displays of support. The College of Science, the Hesburgh Libraries, the Boler-Parseghian Center for Rare Diseases, and the Patient Advocacy Initiative at the University of Notre Dame are grateful to join others around the world to show support for those affected by a rare disease. Rare Disease Day is a global effort that raises awareness for the 300 million people worldwide living with a rare disease, their families, and their caregivers. 4:30 – 6:45 pm | VIP Welcome & “Setting the Scene” Pick up your Rare Disease Day credentials and enjoy family-friendly, star-themed activities, including a “Hollywood Wall of Fame,” star-shaped cookie decorating, face painting, a “Paparazzi” photo station to strike a pose on the red carpet, and a scavenger hunt.

Original title: “Rare Disease Day 2026: Rare Stories, Real Stars - Rare Disease Day 2026”

The post International Childhood Cancer Day Policy Event  appeared first on EURORDIS-Rare Diseases Europe.

Original title: “International Childhood Cancer Day Policy Event ”

At Channeling Hope, we are gearing up to celebrate Rare Disease Day 2026. Rare Disease Day is a globally coordinated movement dedicated to people living with rare diseases to advocate for equity in social opportunities, healthcare, and access to diagnosis and therapies. Learn how to take action for Rare Disease advocacy ... Click the EveryLife Foundation Rare Disease Week button below to register for these free events dedicated to advocating for policies to advance innovation in rare disease care and research: Building an international rare disease community is part of our mission, and on February 28th, we celebrate NALCN champions and all Rare Disease advocates in raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families, and caregivers. Want to celebrate Rare Disease Day with your child and their schoolmates? Check out these Rare Disease Day coloring pages and infographics. Or reach out to Emily ([email protected]) for help creating a flyer specific to your family (see Kam's example below). At the 2nd NALCN Channel & Related Disease International Conference in October 2025, we launched the Raising Hope Capital Campaign to raise $2.8 million by 2028 to move an effective treatment through clinical trials and safely to our community. This Rare Disease Month, we're seeking more CHF Champions to start fundraisers and spread the word to amplify CHF's mission to advance care and develop treatments for individuals affected by NALCN Channel-related diseases.

Original title: “Ready and Raring for Rare Disease Day 2026”

We explain why a European Blueprint is needed, and how we'll be convening our community to develop it. The post Developing a European Blueprint for Rare Diseases: How this work will unfold appeared first on EURORDIS-Rare Diseases Europe.

Original title: “Developing a European Blueprint for Rare Diseases: How this work will unfold”

Dr. Suneel Kamath addresses patient concerns on the topic of clinical trial participation in the gastrointestinal cancer treatment space. So, while it certainly feels risky and scary in many ways, it is often a great way for people to get access to newer therapies before they are otherwise available. I also emphasize that being in a clinical trial is never a coercive situation. If at any point it no longer makes sense for you, you can pull out at any time. Generally, these days, we are designing these trials in a great way where there is usually some sort of biomarker or a good rationale for why a drug will work in a certain disease or for a certain person. The biggest thing I always try to emphasize for people regarding clinical trials in the cancer space is that you are never going to be a "guinea pig." That is often a fear many people have, and it is very understandable. In cancer clinical trials, active treatment is always given; patients receive at least the routine standard of care that we provide today. You are not going to get less treatment than you should; there is really only the opportunity to potentially get more. I would view it through that lens, that it is a potential advantage, really. The other thing I would emphasize is that while it might feel very new or like you are taking a big risk, I always like to remind people that every medication we use today was originally studied in a clinical trial. During the discussion, he explained what he wants patients to understand about participating in clinical trials for their gastrointestinal cancers. Additionally, he imparted key updates from this years · 2026 ASCO Gastrointestinal Cancers Symposium in another article from CURE! How do you address patient concerns on the topic of clinical trial participation?

Original title: “Why Clinical Trials May Offer More for Patients With GI Cancers | CURE”

Original title: “Office of the Director, Notice of Charter Renewal”

Original title: “Situs Inversus: When the Body’s Map Is Reversed”

Be a part of MassBio’s 2026 Rare Disease Day, when our community comes together with rare disease patients, caregivers, advocates, and researchers to raise awareness and celebrate advancements.

Original title: “2026 Rare Disease Day Forum - MassBio”

The post Premiere of ‘No One Rides Alone’ appeared first on EURORDIS-Rare Diseases Europe.

Original title: “Premiere of ‘No One Rides Alone’”