C.D. Leganés is carrying out during ... the Spanish Federation of Rare Diseases (FEDER), to promote the visibility of the needs of people affected by these pathologies. As a highlight of this campaign, the club wanted to take part in the World Day for Rare Diseases, which was ... C.D. Leganés is carrying out during this season 2023/24 the campaign "Rare diseases, unique people" in collaboration with the Spanish Federation of Rare Diseases (FEDER), to promote the visibility of the needs of people affected by these pathologies. As a highlight of this campaign, the club wanted to take part in the World Day for Rare Diseases, which was celebrated on February 29th, with various actions aimed at raising funds for research and the search for possible treatments. In addition, the Federación de Peñas of the club participated in the auction, obtaining the shirt of captain Sergio González, which will be framed and displayed in the Peñista Space located at the Estadio Municipal Butarque. Additionally, the Peñas Federation will donate to FEDER another €3,600 from the sale of charity scarves with the slogan "Rare diseases, unique people".

Original title: “Leganes gets involved in the campaign "Rare diseases, unique people". - C.D. Leganés Foundation”

Every rare disease community deserves a curated trial finder. The barriers to building them have dropped substantially. Trial navigation is itself a clinical access problem, one that should receive the same care and thoroughness we bring to developing the therapies those trials are designed to test. Clinical trial navigation is treated as an awareness problem, but in rare cancers the tools themselves are the barrier. See what a subtype-level finder fixes. But none of these efforts have fully solved the subtype-level navigation problem for rare lymphoma subtypes, partly because the source data in ClinicalTrials.gov is not structured in a way that supports it. What would a purpose-built solution look like? It would organize trials by specific disease subtype, not broad diagnostic categories. It would provide plain-language summaries alongside technical details. It would allow filtering by treatment stage, newly diagnosed versus relapsed or refractory, because patients at different points in their treatment journey need different trials. There is no prioritization to help distinguish a landmark phase 3 randomized trial from a small phase 1 dose-escalation study at a single academic center. This is not a criticism of ClinicalTrials.gov. It serves its intended purpose as a comprehensive registry. But a registry is not a navigation tool, and we have been treating it as both. The gap is particularly significant in rare cancers for two reasons. First, the signal-to-noise ratio is worse. A search that returns 300 results for a disease affecting 9,000 patients per year will contain a high proportion of tangentially relevant listings. The solutions proposed tend to follow accordingly: educate patients, remind physicians, mandate trial discussions as part of standard care, embedded directly within NCCN treatment algorithms. According to a landmark landscape report by the American Cancer Society Cancer Action Network (ACS CAN), a substantial portion of eligible cancer patients never enroll simply because their doctor never brought it up. When patients actually are asked to participate, more than 50 percent agree to enroll. The bottleneck is rarely patient refusal; it is the lack of an initial provider offer.

Original title: “Clinical trial navigation is a clinical access problem”

Last month’s FDA advisory committee meetings for therapies developed by Capricor Therapeutics and Replimune effectively illustrate the different outcomes for patients when the agency prioritizes specialization in its reviews.

Original title: “Capricor, Replimune illustrate why FDA must listen to rare disease specialists”

facebook as a lifeboat an interview study exploring the role of facebook in supporting parents of children with rare diseases

Original title: “Facebook as a Lifeboat: An Interview Study Exploring the Role of Facebook in Supporting Parents of Children with Rare Diseases.”

Earlier in his career, he led global ... of rare-disease therapies, with earlier commercial leadership roles at Acorda Therapeutics, Takeda Pharmaceuticals and Johnson & Johnson. Mr. Benecchi holds an MBA from Duke University's Fuqua School of Business and a BA from Colby College. ... Inventiva is a clinical-stage biopharmaceutical company focused on the research and development of an orally administered small molecule for the treatment of patients ... Earlier in his career, he led global launch readiness for UCB's BIMZELX® (bimekizumab) and, at Alexion, directed global commercial excellence and launch readiness across a portfolio of rare-disease therapies, with earlier commercial leadership roles at Acorda Therapeutics, Takeda Pharmaceuticals and Johnson & Johnson. Mr. Benecchi holds an MBA from Duke University's Fuqua School of Business and a BA from Colby College. ... Inventiva is a clinical-stage biopharmaceutical company focused on the research and development of an orally administered small molecule for the treatment of patients with MASH. Daix (France), New York City (New York, United States), August 31, 2026 – Inventiva (Euronext Paris and NASDAQ: IVA) (“Inventiva” or the “Company”), a clinical-stage biopharmaceutical company focused on the development of an oral therapy for the treatment of metabolic dysfunction-associated steatohepatitis (“MASH”), today announced the appointment of Chris Benecchi as Chief Operating Officer. The Company is currently evaluating lanifibranor, a novel pan-PPAR agonist, in the NATiV3 pivotal Phase 3 clinical trial for the treatment of adult patients with MASH, a common and progressive chronic liver disease. Lanifibranor is an investigational medicine and has not been approved for use by any regulatory authority. These statements include, but are not limited to, forecasts and estimates with respect to Inventiva’s NATiV3 Phase 3 clinical trial with lanifibranor in patients with MASH, including the quality of trial results, design, duration, timing, costs, and funding, timing of clinical trial data releases and publications, the information, insights and impacts that may be gathered from clinical trials, the potential therapeutic benefits of lanifibranor, potential regulatory submissions, approvals and commercialization, Inventiva’s pipeline and development plans, and Inventiva's future activities, expectations, plans, growth and prospects.

Original title: “Inventiva Appoints Chris Benecchi as Chief Operating Officer”

Over 500 advocates from 47 states, Puerto Rico, and D.C. participated in 265 meetings with their Members of Congress!

Original title: “More than 500 Advocates Participated in RARE Across America 2026”

Experiences from Georgia and Portugal show how patient organisations and teachers are adapting the Rare Disease Day School Toolkits to their own communities, what they… Continue reading How schools are bringing rare disease awareness to life Bringing rare diseases into schools is about more than awareness on one day. By introducing these conversations into the classroom early, we can help children grow up understanding and valuing difference, laying the foundations for a more inclusive society and a more equitable future for the rare disease community. The Rare Disease Day School Toolkits were created to help teachers, families and patient organisations bring rare diseases into those conversations in an engaging and age-appropriate way, with empathy and inclusion at their heart. Its President, Anadá Filipitsch Gomes, approached the headteacher of Bairro Operário School, attended by her daughter Constança, who lives with KAT6A syndrome. Together, they developed a programme to mark Rare Disease Day. At the heart of it was the opportunity for pupils to meet a family living with a rare disease. Constança’s mother visited every class from year 1 to year 4, talking about Rare Disease Day, KAT6A syndrome and her daughter.

Original title: “How schools are bringing rare disease awareness to life | Rare Disease Day 2026”

My CSF Leak, IIH & VP Shunt Journey My Story, My Life, My Awareness Message MaggieBrisbane, Queensland, Australia31 years old Introduction My journey did not… Continue reading story of my life

Original title: “story of my life - Rare Disease Day 2026”

In this episode of Denatured, you’ll hear from Hilary Rosselot, executive director at the National Fragile X Foundation and Jordi Fàbrega, co-founder & CEO at CONNECTA Therapeutics. From trial endpoints and placebo effects to neuroplasticity modulation, we explore what it will take to build a sustainable path to the first approved therapy.

Original title: “Fragile X at a turning point: Breaking through the clinical bottleneck”

Limited opportunities and burnout are just two reasons biopharma professionals stop looking for work, with some pivoting away from the industry or turning to consulting. Two career coaches discuss why ending a job hunt is harmful and what to do instead.

Original title: “Nearly 1/4 dealing with long-term unemployment call it quits on biopharma job searches”

My name is Esteban, I am 17 years old, and I live in Colombia. I am a second‑semester medical student, and my dream is to… Continue reading Fighting for My Future — and the Lives of Many Others Like Me I am a second‑semester medical student, and my dream is to become a physician who advocates for patients with rare diseases—patients like me, and many others whose voices are often unheard.I live with Late-Onset Pompe Disease, a rare genetic condition that requires strict and uninterrupted enzyme replacement therapy (ERT) every two weeks. It doesn’t. Every Pompe patient needs absolute continuity. Interruptions are not just harmful—they are life‑altering.I want to keep studying. I want to keep walking. I want to keep dreaming. And I want the same for every person living with Pompe disease or any rare condition that depends on continuous treatment.I am sharing my story because visibility creates change. I hope that by raising my voice, I can protect my health, continue my education, and help build a future where rare disease patients in Colombia receive the care they deserve — consistently, compassionately, and without interruption.

Original title: “Fighting for My Future — and the Lives of Many Others Like Me - Rare Disease Day 2026”

I was seeing my local neurologist for issues. I thought I had MS but he believed it was all in my head and just kept… Continue reading Finally Getting Answers So around 2019 we ask for referral to a neurologist and a medical student diagnosed me with this disease – IVIG.

Original title: “Finally Getting Answers - Rare Disease Day 2026”

In this episode of Denatured, you’ll hear from Ian McGowan, Chief Medical Officer at Synklino and James Hunter, associate professor of transplantation at Oxford University. We explore transplantation and antiviral innovation and why CMV remains one of the most persistent and consequential challenges facing transplant patients today.

Original title: “Rewriting the rules of CMV prevention”

Uneven access to early detection and the need for stronger European cooperation. The post A Lottery at Birth: Why the EU needs a shared approach to newborn screening appeared first on EURORDIS-Rare Diseases Europe.

Original title: “A Lottery at Birth: Why the EU needs a shared approach to newborn screening”